How to Execute White-Space Patent Research with PatSnap as Exemplified using the CRISPR Gene Editing Technology (2023)

James H. Moeller - Moeller Ventures, LLC -

October 10, 2022

This report presents a tutorial on executing patent document white-space research using the PatSnap Connected Innovation Intelligence platform. This is a follow-on to the patent landscaping tutorial report (A Patent Landscaping Tutorial Using the PatSnap Analysis Tool and CRISPR as the Focus Technology) and will also use CRISPR as the focus technology. This report was also motivated by the 2021 National Institute of Health (NIH) project as part of providing commercialization assessments to SBIR (Small Business Innovation Research), and STTR (Small Business Technology Transfer) funded companies. The NIH project was executed for a contract awarded to Vikriti Management Consulting, where Moeller Ventures served as a subcontractor executing 200 patent landscapes for 164 life science companies.

Developing intellectual property is difficult. It usually involves significant R&D followed by the pursuit of patents via an expensive prosecution process that’s fraught with approval uncertainties. Leveraging white-space research into IP development has historically been expensive, problematic, and error-prone. It is, after all, the process of searching an IP sector for topic areas that are either lightly patented or void of any patent filings. This roughly equates to the paradoxical challenge of searching for IP that’s not present.

However, new analytics tools are dramatically changing the white-space research process by leveraging semantic analysis, natural language processing (NLP), and similarity assessment to identify landscape IP clusters as well as white-space IP voids. These processes can add significant value in analyzing a patent document domain collection, visualizing it on a landscape diagram, and querying the domain space for patenting opportunities. Furthermore, these automated techniques can be applied iteratively to gain significant insights into IP development.

Across the 164 companies and 200 patent landscapes provided in the NIH project, only a few companies seemed to understand the value of executing white-space research and its potential to augment the company's IP development process. This was surprising given the significant extent to which nearly every company relies on IP and patents to protect its inventions and market differentiating advantages. Ultimately white-space research can be used to focus R&D initiatives and draft better patent applications that avoid crowded landscape areas and improve the chances of having the patent approved.

The five steps of the white-space research process covered in this report are listed below.

Step 1: Determine the Context of the White-Space Research

Step 2: Create the Domain Collection of Patent Documents

Step 3: Generate the Landscape Diagram

Step 4: Query the Landscape Diagram

Step 5: Iterate the White-space Research Process

(Video) How to develop and maintain a world class patent portfolio 1

Inquiries regarding custom patent and intellectual property analysis using the PatSnap Connected Innovation Intelligence platform can be directed to the following individuals.

Step 1: Determine the Context of the White-Space Research, i.e., “CRISPR Nuclease”

Determining the context of the white-space research is essential in establishing the patent document query that will be used to create the domain collection for the analysis. This step typically requires a relatively high level of detailed insight into the topic area on which the white-space research will be focused and is often directly linked to development initiatives that initially motivate the inquiry. For example, a business may have a product development project where it believes it is establishing patentable IP, and it uses a specific set of keywords and phrases to create a white-space analysis domain collection. Alternatively, maybe it’s a university research initiative that has been granted a patent in a specific area, and it uses a semantically similar patent document query to create a domain collection that can be searched for other nearby patenting opportunities.

For this CRISPR white-space research example, it is helpful to start with some cursory background on the CRISPR gene editing technology that can provide insight into an approach for an instructive white-space analysis. CRISPR is broadly known as the biological technology that enables the editing of DNA (deoxyribonucleic acid), which makes up the genetic material of all living things. Since the invention of the CRISPR gene editing technology in 2012, patenting activities in this area have increased dramatically across a wide variety of complex topics, with each topic potentially an area that can be explored for white-space patenting opportunities. However, those topics all likely require significant expertise to determine the context of the analysis and are thus not good instructive examples. But a more fundamental examination of CRISPR reveals a more straightforward white-space research example: an analysis focused on the CRISPR nuclease.

CRISPR is named for the repeating pattern of nucleotides found in a specialized region of the DNA backbone. The acronym is derived from the phrase “Clusters of Regularly Interspaced Short Palindromic Repeats.” Nucleotides are organic molecules that are essentially the building blocks of DNA and RNA. However, the CRISPR gene editing technology is often described as having two components, a guide RNA and a nuclease protein. The guide RNA is similar to a shorter segment of DNA, but it can be programmed to search for and recognize specific sequences or genes within a DNA strand. This is combined with a nuclease protein responsible for executing some action on the DNA strand. For example, one of the initial and more popular nucleases associated with CRISPR is Cas9, which performs the action of cutting out the targeted gene. Various additional types of nucleases have also been associated with CRISPR, including Cas3, Cas12, Cas13, CRISPR-Csm, and others. Each nuclease can act on DNA (and RNA) in different ways. CRISPR nuclease research is one of the more active areas producing additional fundamental patents for editing genetic material. As a result, an instructive CRISPR white-space analysis can focus on these nuclease developments and simply utilize the various nuclease names to exemplify the white-space research.

Step 2: Create the Domain Collection of Patent Documents

Armed with the context of the white-space research, the next step is to create the domain collection of patent documents that will be used in the analysis. For white-space research, in particular, it is desirable to make the domain collection as broad as possible, given the context constraints. After all, white-space research is focused on finding new patenting opportunities. Any relevant references can potentially be prior art barring new patents, regardless of whether those references are in any section of pending applications, active granted patents, abandoned applications or patents, or expired patents.

Various techniques can create a domain collection specifically for white-space research. For example, keywords and key phrases can be used as queries into worldwide patent databases to find relevant documents representing topics or technology sectors. This type of text query can match specific text in a patent document title, abstract, description, or claims, with keywords and phrases matched across all sections. In addition, domain collections can be created by applying machine learning (ML) and natural language processing (NPL) techniques to identify semantically similar patent documents to a seed document or a set of seed documents. Most patent analytics services offer some type of NPL-enabled semantic search capability that enables users to create domain collections via this technique.

This CRISPR white-space research example will focus on the nuclease protein of the gene editing capability. As a result, the patent document query will be kept simple and utilize a keyword query using the keywords “CRISPR” and “nuclease.” Within PatSnap, the search can be executed either by the Simple search or the Field search options, but either way, it is essential to perform the search over all sections of the patent documents (title, abstract, description, and claims). The Simple search defaults the keyword query to all sections, whereas the Field search requires selecting “Title/Abstract/Claims/Description” from the text search options. Entering the keywords “CRISPR” and “nuclease,” not enclosed by quotes, returns 49,905 patent documents representing 14,002 simple patent families. This query looks too broad as it returns many documents where the keywords are used independently. Entering the phrase “CRISPR nuclease,” enclosed in quotes, returns a much more manageable domain collection of 3,374 patent documents representing 838 simple patent families and more likely focuses the patent documents specifically on those nucleases directly associated with CRISPR. Once the search results are generated, the patent documents can be saved to a workspace within PatSnap. Finally, it’s also worth noting that the domain collection results may not include the most recent patent filings due to publication delays for applications, such as the 18-month publication delay for USPTO-filed applications.

Step 3: Generate the Landscape Diagram

Before generating the landscape diagram, it’s essential to ensure that the patent documents are grouped with only one representative document per simple patent family. This grouping will eliminate duplicates of the same patent document filed in various worldwide jurisdictions and make the white-space research more straightforward to understand via the queries of step 4. Within PatSnap, the grouping setting is accessed under “Data Management” in the top-middle menu when viewing the tabular representation of the domain collection. Select “One representative per simple family” in the provided drop-down.

To generate the landscape diagram, select the “Analysis & Alerts” menu item from the top-middle menu of the tabular domain collection view and then select the “Landscape” option. This will open a new tab within your browser and display the default view of the landscape topography diagram (see Exhibit 1). This landscape topography is created by semantically grouping the domain collection patent documents. These groupings are shown on the topography according to the concentrations of semantically similar documents, with the high-level groupings positioned on the topography in a relatively semantically similar arrangement. The topography's peaks and valleys represent the document concentrations in the domain collection. For example, the white areas, like mountain peaks, represent the topic areas with the highest document concentrations. Successively lower document concentrations are represented by gray, dark green, lighter green, and light yellow. A light blue area represents an area completely lacking any documents. The high-level groupings can be shown via the “Select Grid(s)” option in the lower right corner, which shows the landscape grid and indicates each high-level semantic grouping via different shaded colors (see Exhibit 2).

The default landscape diagram also shows one keyword/key phrase callout textbox for each high-level grouping of the patent documents. These keywords or phrases are derived from the NPL analysis of the text of the patent documents in the high-level grouping and are designed to indicate the general grouping topic content. It’s often visually convenient to format and arrange these textbox callouts in preparation for the white-space analysis. Black backgrounds can be added to the text boxes via the “Setting” menu item to the left. The position of the textbox callouts can be adjusted via the “ScreenShot” option in the lower right corner. For the CRISPR nuclease analysis, the resulting landscape diagram is shown in Exhibit 1.

Finally, additional textbox callouts can be added on a per-grid basis via the left sidebar “Setting” option and the “Edit Label” capability. Adding additional callouts on a per-grid basis can sometimes help identify the high-level keywords or phrases in areas of interest, such as high-concentration areas or potential white-space areas. However, these additional high-level textbox callouts often lack the detail to provide real insight into potential patenting opportunities, as the keywords and phrases for each grid box are primarily derived from the higher-level grouping with only slight differences between grid blocks within a grouping.

Exhibit 1: “CRISPR Nuclease” Default Landscape Diagram

How to Execute White-Space Patent Research with PatSnap as Exemplified using the CRISPR Gene Editing Technology (1)

Exhibit 2: “CRISPR Nuclease” Default Landscape Diagram with Shaded Groupings

How to Execute White-Space Patent Research with PatSnap as Exemplified using the CRISPR Gene Editing Technology (2)

Step 4: Query the Landscape Diagram

The topographical landscape diagram can now be queried with keywords and key phrases to visually indicate the location and quantity of matching patent documents. Iterative keyword / key phrase queries can refine the search to reveal the topics that are either infrequently addressed, contained within more unique patent documents, or not addressed at all. This is essentially a visually interactive process of querying what’s present in the domain collection to gain insights into what’s not present or only lightly represented. The intelligent selection of domain-specific keywords and key phrases defines this search process, which is why this type of white-space research typically requires significant expertise in the research domain area.

Within PatSnap, the principal mechanism used to query the landscape diagram is the Search function in the left sidebar menu. This function has two modes of keyword / key phrase entry, a “Simple” search input and a “Field” search input. The Simple search uses any entered keywords or key phrases to query across all sections of every patent document in the domain collection. Any document matching the query will be shown on the landscape diagram as a gray dot. Specific key phrases are created by enclosing the relevant keywords in quotes. PatSnap’s Boolean search capabilities can also be used in the search input field. The Field search option works similarly, except that it allows the query to be further filtered by searching only in certain document sections (title, abstract, description, or claims) or by specifying other metadata about the patent documents (i.e., dates, assignees, inventors, etc.). While the Field search option is quite powerful, this white-space research example will only need to utilize the broader Simple search mechanism. After all, white-space research focuses on finding new patenting opportunities, which requires searching all sections of the patent documents in the domain collection that may contain prior art that limits or prohibits new patents.

As a simple initial example, Exhibit 3 shows the results when “cas9” is entered into the Simple search field. The landscape diagram becomes covered with gray dots. As a result of selecting the grouping option of “one representative per simple family,” each dot represents a unique patent document that includes “cas9” in either the title, abstract, description, or claims text. The high number of gray dots indicates that the Cas9 nuclease is a relatively thoroughly patented topic within this domain collection. This isn’t surprising since it is one of the initial and most widely developed nucleases associated with CRISPR. But as a result, finding white-space patenting opportunities with the Cas9 nuclease would likely require significant detailed research.

Exhibit 3: Landscape Query for “cas9”

How to Execute White-Space Patent Research with PatSnap as Exemplified using the CRISPR Gene Editing Technology (3)

The location of the dots on the landscape diagram can also provide insightful information. In particular, dots that have landscape locations on or near high concentrations areas of the landscape (areas represented by the peaks on the topography) are potentially less unique. These patent documents have a higher risk of encountering more comparable and competitive IP and increased freedom-to-operate (FTO) issues. Conversely, patent documents located in low document concentration areas (the valleys on the topography) are potentially more unique with lower competitive risks and FTO limitations. As a result, query tactics focusing on the lower concentration areas of the landscape topography have a higher likelihood of uncovering white-space patenting opportunities.

It is also instructive to note that the gray dots matching the search query are rendered on the diagram as mouse-over and interactive hyperlinks. Moving the cursor over a gray dot will automatically show a pop-up display of the patent document publication number and the document title and assignee. This is exemplified in Exhibit 4 for an international patent application (WO2021151065A3) filed by The General Hospital Corporation, more commonly known as Massachusetts General Hospital. This looks to be a relatively unique patent document with a placement in a topographical valley, which might merit more detailed research to determine why it is identified as being so unique. These pop-ups are very convenient for quickly scanning the documents in areas of interest on the landscape.

Exhibit 4: “cas9” Landscape with General Hospital Corporation Patent Document Highlighted

How to Execute White-Space Patent Research with PatSnap as Exemplified using the CRISPR Gene Editing Technology (4)

In addition, clicking on a gray dot will open a pop-up window that shows the full patent document in a tabbed format and provides links to download the patent document or open it in a separate window. This is exemplified in Exhibit 5 for the Massachusetts General Hospital patent application noted above. These pop-up windows are additional conveniences enabling more detailed review of patent documents on the landscape to quickly find and review the context of the matching keywords or key phrases and review the specific patent claims to develop an understanding of what’s represented in the invention. Understanding what’s already represented in the landscape diagram is a critical step in identifying what’s not represented and what could potentially be white-space patenting opportunities.

Exhibit 5: “cas9” Landscape with General Hospital Corporation Patent Document Clicked

How to Execute White-Space Patent Research with PatSnap as Exemplified using the CRISPR Gene Editing Technology (5)

As an additional example, “cas13” is entered in the Simple search field. Cas13 is a more recently discovered nuclease protein that seems to target RNA instead of DNA and can be used for identifying viruses. Exhibit 6 shows that landscape diagram with a notably lower number of gray dots designating the matching patent documents. This indicates a less thoroughly patented environment with potentially more new patenting opportunities. Again, reviewing the patent documents shown in the valleys of the topographical diagram can often lead to insights regarding unique patenting topics.

Exhibit 6: Landscape Query for “cas13”

How to Execute White-Space Patent Research with PatSnap as Exemplified using the CRISPR Gene Editing Technology (6)

PatSnap also provides an additional technique to analyze specific portions of the landscape diagram in more detail via its “Select Grid(s)” option available in the lower right corner of the diagram. Exhibit 2 showed the landscape with this select-grids capability activated. This shows the high-level groupings of the patent documents and the fainter background outlines of the grids on the diagram. The landscape diagram is essentially partitioned via horizontal and vertical grid squares. These squares can be selected so that the patent documents in the selected areas can be further analyzed by either exporting those specific patent documents or by computing a new landscape utilizing only the documents in the selected area.

For example, the landscape diagram shows a significant topographical valley in the lower left quadrant. This is potentially a white-space patenting opportunity area that needs further analysis. To do this, the grid squares covering this valley can be selected, as shown in Exhibit 7. The display indicates that nine grid squares have been selected, with 59 patent documents included in that selected area. Via the “View Patents” option in the lower right corner, these patent documents can be viewed in a list format and exported in a spreadsheet format for further examination. In addition, via the “New Landscape” option, a new landscape can be computed using only the documents from the selected grids. This new landscape is shown in Exhibit 8 and can be further explored via the same interactive querying process described here in step 4. Note that this new landscape diagram isn’t simply a zoomed-in image of the original landscape section but rather a newly computed landscape using the selected 59 patent documents as a new domain collection.

Exhibit 7: Landscape with Valley Grid Squares Selected

How to Execute White-Space Patent Research with PatSnap as Exemplified using the CRISPR Gene Editing Technology (7)

Exhibit 8: New Landscape from 9 Selected Grid Squares

How to Execute White-Space Patent Research with PatSnap as Exemplified using the CRISPR Gene Editing Technology (8)

As a final query example, “csm complex” is entered into the Simple search field. This key phrase is intended to represent a very recent development from Dr. Jennifer Doudna’s lab at the University of California, Berkeley, where multi-protein CRISPR-Cas effector complexes were shown to target RNA more effectively than the Cas13 nuclease. This was posted to the bioRxiv website on June 20, 2022, via a report entitled “Precise Transcript Targeting by CRISPR-Csm Complexes” and announced via the Doudna Lab Twitter feed (@doudna_lab) on June 21, 2022.

The landscape diagram for this query is shown in Exhibit 9 and indicates only six matching patent documents; three from Caribou Biosciences, one from the University of California Berkeley, and two from joint assignees North Carolina State University and BASF Plant Science Company GMBH. The patent application document specifically pertaining to the June 20, 2022, UC Berkeley announcement is not included in this landscape due to the publishing delay associated with new application filings. The low number of matching patent documents indicates the early stage of IP developments in this area as well as the potential for new patenting opportunities.

Exhibit 9: Landscape Query for “csm complex”

How to Execute White-Space Patent Research with PatSnap as Exemplified using the CRISPR Gene Editing Technology (9)

This situation, where the new UC Berkeley development isn’t yet included in the available patent documents, can be used to exemplify another PatSnap capability that’s useful for white-space research: the placement of free-form text descriptions on the landscape diagram. This capability is quite useful when a landscape has been created, and a text description of an innovation is semantically placed on the landscape to determine the description’s relative landscape position and its proximity or similarity to other patent documents in the domain collection. This technique of entering text descriptions can be used iteratively to refine ongoing R&D as well as assess competitive and FTO issues for new innovations.

Within the PatSnap tool, the text description entry capability can be found via the “Text Analysis” option on the left sidebar of the landscape diagram. This option allows for the entry of a title and a text description, after which the tool computes the semantic location of the entered text on the landscape diagram. For example, if the title and abstract of the UC Berkeley bioRxiv published report are entered, PatSnap computes a landscape location of that entered text as indicated by the red triangle in the lower right corner of the landscape diagram shown in Exhibit 10. This placement may require additional research as it is near a high patent document concentration area. But it is indeed not located near any of the other “csm complex” matching documents, indicating some level of uniqueness within the subdomain identified by that query.

Exhibit 10: Landscape with UC Berkeley Report Semantic Placement

How to Execute White-Space Patent Research with PatSnap as Exemplified using the CRISPR Gene Editing Technology (10)

Step 5: Iterate the White-space Research Process

The final step in this white-space research process is simply the iterative application of the preceding steps. This includes executing additional queries on the landscape diagram, selecting alternative grid partitions for detailed analysis, placing other experimental text descriptions on the landscape, or even generating new alternative domain collections to analyze. This is one of the most powerful advantages of applying data science and analytics to the white-space research process, as all of these can be executed quickly in an iterative fashion.

It's sometimes argued that the application of data science techniques to white-space research can produce slightly suboptimal results. This is usually associated with the techniques used to derive patent document similarities (semantic analysis, natural language processing, and machine learning), as well as the plotting of the document similarity clusters on a topographical landscape diagram and the use of this derived landscape to query for white-space patenting opportunities. While automated analysis techniques can result in some inaccuracies, today’s text similarity processes are dramatically better and, in particular, substantially faster than any classical white-space search techniques. So, the additional capability to quickly iterate the steps and techniques described in this report can add significant value that far outweighs any inaccuracies resulting from the application of data science to derive document similarities. The application of data science to processes like white-space research is and will continue to be the gold standard for the derivation and extraction of business intelligence from big data repositories such as patent documents.

About the Author: Jim Moeller provides customized consulting services leveraging analytics and data science tools for data integration and intelligence mining, aimed at projects focused on intellectual property research, market analysis, and business development. Executed project domains have covered medical devices, pharmaceuticals, biotechnology, digital and connected health, wearables, telemedicine, IoT (Internet-of-Things), and wireless communications. Jim is a U.S. Registered Patent Agent with experience spanning consulting, investment banking, and engineering.


How is Crispr gene editing done? ›

CRISPR/Cas9 edits genes by precisely cutting DNA and then letting natural DNA repair processes to take over. The system consists of two parts: the Cas9 enzyme and a guide RNA. Rapidly translating a revolutionary technology into transformative therapies.

Who has the patent for Crispr gene editing? ›

CRISPR Therapeutics, Intellia Therapeutics and Caribou Biosciences Announce Grant of U.S. Patent for CRISPR/Cas9 Genome Editing. ZUG, Switzerland and CAMBRIDGE, Mass.

Can you patent CRISPR? ›

In the United States, Broad has been allowed or granted 31 CRISPR patents, including 26 patents for CRISPR-Cas9, as well as 3 for CRISPR-Cas12/Cpf1. The USPTO has also granted patents directed to CRISPR-Cas9 to UC Berkeley (UCB), University of Vienna and Emmanuelle Charpentier.

How do you conduct a CRISPR experiment? ›

  1. Step 1: Design the CRISPR sgRNA. The first step in your CRISPR experiment is to design the customizable guide RNA to target your DNA sequence. ...
  2. Step 2: Edit DNA Precisely with CRISPR. ...
  3. Step 3: Analyze Data from CRISPR Experiment.

What are the three stages of CRISPR? ›

The CRISPR-Cas system acts in a sequence-specific manner by recognizing and cleaving foreign DNA or RNA. The defence mechanism can be divided into three stages: (i) adaptation or spacer acquisition, (ii) crRNA biogenesis, and (iii) target interference (figure 1).

What is CRISPR and how is it used? ›

CRISPR is a highly precise gene editing tool that is changing cancer research and treatment. Credit: Ernesto del Aguila III, National Human Genome Research Institute. Ever since scientists realized that changes in DNA cause cancer, they have been searching for an easy way to correct those changes by manipulating DNA.

How is gene editing performed? ›

Gene editing is performed using enzymes, particularly nucleases that have been engineered to target a specific DNA sequence, where they introduce cuts into the DNA strands, enabling the removal of existing DNA and the insertion of replacement DNA.

How is CRISPR being used today? ›

“[CRISPR is] a tool that scientists and clinicians around the world are using to understand our genetics, the genetics of all living things, and — most importantly — to intervene in genetic disease.”

Who owns the patent rights to CRISPR? ›

Feb 28 (Reuters) - A U.S. tribunal overseeing patent disputes ruled on Monday that patents on the breakthrough gene-editing technology known as CRISPR belong to Harvard University and the Massachusetts Institute of Technology.

How many CRISPR patents are there? ›

There are now more than 11,000 families of patents on CRISPR-related technologies, according to the business-intelligence firm Centredoc in Neuchâtel, Switzerland.

What company is leading in CRISPR technology? ›

Intellia Therapeutics surged to the top spot among CRISPR stocks in 2021. The company and its partner, Regeneron (NASDAQ:REGN), announced impressive interim results in June 2021 from a phase 1 study evaluating NTLA-2001 in treating rare genetic disease transthyretin (ATTR) amyloidosis.

Is CRISPR legal? ›

CRISPR is legal in the US. Many hospitals and biotech companies are currently pursuing clinical trials with CRISPR. These trials are regulated by the FDA. If the trials are successful then the FDA will grant these organizations approval to market the drug as a commercial product.

Is CRISPR copyrighted? ›

A tribunal within the US Patent and Trademark Office has ruled that CRISPR is an intellectual property of MIT and Broad Institute of Harvard. The University of California Berkeley and University of Vienna are other institutions who claim that CRISPR is their intellectual property.

Can altered genes be patented? ›

The Supreme Court's ruling did allow that DNA manipulated in a lab is eligible to be patented because DNA sequences altered by humans are not found in nature. The Court specifically mentioned the ability to patent a type of DNA known as complementary DNA (cDNA).

Is CRISPR easy to use? ›

A big concern is that while CRISPR is relatively simple and powerful, it isn't perfect. Scientists have recently learned that the approach to gene editing can inadvertently wipe out and rearrange large swaths of DNA in ways that may imperil human health.

What are the benefits of CRISPR? ›

What are the advantages of CRISPR over other genome editing tools? The CRISPR-Cas9 system can modify DNA with greater precision than existing technologies. An advantage the CRISPR-Cas9 system offers over other mutagenic techniques, like ZFN and TALEN, is its relative simplicity and versatility.

How do you knock out a gene with CRISPR? ›

How CRISPR gene knockout works. A CRISPR-associated (Cas) enzyme is used to cleave target DNA, resulting in a double-strand break (DSB). The Cas enzyme is directed by the guide RNA (gRNA) to a user-defined site in the genome, and then the Cas enzyme cuts the DNA.

How does CRISPR work for dummies? ›

CRISPR (pronounced crisper), is a powerful DNA or gene-editing tool whose origin lies in the natural adaptive immunity of bacteria. It enables DNA to be cut at precise locations, allowing for its accurate and targeted renewal or replacement.

How many types of CRISPR are there? ›

Three major types of CRISPR-Cas systems are at the top of the classification hierarchy. The three types are readily distinguishable by virtue of the presence of three unique signature genes: Cas3 in type I systems, Cas9 in type II, and Cas10 in type III [5].

How does CRISPR cut DNA? ›

The CRISPR-Cas9 system consists of two key molecules that introduce a change (mutation?) into the DNA. These are: an enzyme? called Cas9. This acts as a pair of 'molecular scissors' that can cut the two strands of DNA at a specific location in the genome so that bits of DNA can then be added or removed.

Which applications have been developed using CRISPR? ›

Applications of CRISPR
  • Using CRISPR for genome editing.
  • Using CRISPR libraries for screening.
  • CRISPR/Cas9-mediated chromatin immunoprecipitation.
  • Transcriptional activation and repression.
  • Epigenetic editing with CRISPR/Cas9.
  • Live imaging of DNA/mRNA.
  • Therapeutic Applications.

How is CRISPR delivered to cells? ›

The current CRISPR/Cas9 delivery methods include non-viral vectors, viral vectors, and physical delivery. Virus-mediated gene delivery is the most widely used method and it involves integrating CRISPR/Cas9-encoding sequences into the viral genome and releasing the CRISPR/Cas9 gene complex into infected cells.

What are some ways gene editing technology is used? ›

CRISPR has many possible uses, including insert a new gene so the organism produces useful medicines; help treat genetic diseases; create tailor-made organisms to study human diseases; and help produce replacements for damaged or diseased tissues and organs.

What tools are used in CRISPR? ›

Tool NameProvidergRNA suggestion or scoring
CRISPR Targeted Gene DesignerHorizon DiscoveryYes
GuideMakerUnited States Department of Agriculture, Agricultural Research ServiceYes
20 more rows

What equipment is needed for gene editing? ›

The core technologies now most commonly used to facilitate genome editing, shown in Figure 1, are (1) clustered regularly interspaced short palindromic repeats (CRISPR)-CRISPR-associated protein 9 (Cas9), (2) transcription activator-like effector nucleases (TALENs), (3) zinc-finger nucleases (ZFNs), and (4) homing ...

What is the most widely used method of gene editing? ›

The most popular and new method of these is CRISPR, also known as clustered regularly interspaced short palindromic repeats.

How CRISPR will change the world? ›

Thanks to its pinpoint accuracy and relatively low production costs, CRISPR could potentially change everything involving genes: from curing diseases and improving agriculture, to repairing genetic disorders like sickle cell anemia or hemophilia.

What problems can CRISPR solve? ›

Scientists are studying CRISPR for many conditions, including high cholesterol, HIV, and Huntington's disease. Researchers have also used CRISPR to cure muscular dystrophy in mice. Most likely, the first disease CRISPR helps cure will be caused by just one flaw in a single gene, like sickle cell disease.

What are some of the concerns about using CRISPR for gene editing? ›

While CRISPR has the power to cure some diseases, studies have shown that it could lead to mutations that lead to others down the line. If genetic edits are made to embryos, or to egg or sperm cells, these changes will be inherited by all future generations.

Do you need a license to use CRISPR? ›

For academic and non-profit research use, no written license is necessary. For these communities we make CRISPR tools, knowledge, methods and other IP for genome-editing freely available for research.

What companies own CRISPR? ›

Caribou Biosciences and Intellia Therapeutics are associated with the Doudna camp; CRISPR Therapeutics, ERS Genomics and Casebia Therapeutics are associated with Charpentier, and Editas Medicine is associated with Zhang (although notably, Doudna was a co-founder before falling out with Zhang).

What is the potential of CRISPR? ›

The therapeutic potential of CRISPR/Cas9 technology is great, especially in gene therapy, in which a patient-specific mutation is genetically edited, or in the treating of human disorders that are untreatable with traditional treatments.

Who owns the patent? ›

A patent application and any resulting patent is owned by the inventor(s) of the claimed invention, unless a written assignment is made or the inventors are under an obligation to assign the invention, such as an employment contract.

Where was CRISPR first discovered? ›

CRISPR (Clustered Regularly Interspaced Short Palindromic Repeat) sequences were initially discovered in the E. coli genome in 1987, but their function as a safeguard against bacteriophages was not elucidated until 2007.

Who invented CRISPR? ›

Jennifer Anne Doudna ForMemRS (/ˈdaʊdnə/; born February 19, 1964) is an American biochemist who has done pioneering work in CRISPR gene editing, and made other fundamental contributions in biochemistry and genetics.

What biotech company makes CRISPR? ›

CRISPR Therapeutics

The company's mission is "developing transformative gene-based medicines for serious human diseases." CRISPR Therapeutics and its partner, Vertex Pharmaceuticals (NASDAQ:VRTX), hope to be first to market with a CRISPR gene-editing therapy.

What's the stock price of CRISPR? ›

Performance Outlook
Previous Close51.29
Bid50.91 x 1100
Ask51.98 x 2900
Day's Range50.20 - 51.95
3 more rows

Is CRISPR still being developed? ›

The development of CRISPR genome editing opens up new possibilities in precision medicine. Current trials are underway in seven treatment areas: blood disorders, cancers, inherited eye disease, diabetes, infectious disease, inflammatory disease, and protein-folding disorders.

Is CRISPR ethical or unethical? ›

Scientists generally agree that CRISPR-Cas9 should be allowed for use in the creation of human disease models, and in understanding the development and molecular mechanisms of diseases; however, it should be prohibited for the purposes of eugenics or enhancement.

Is CRISPR-Cas9 FDA approval? ›

FDA Approves First Trial Using CRISPR to Correct Sickle Cell Disease Mutation.

When was CRISPR first used for gene editing? ›

CRISPR-Cas9 in the Spotlight

The use of CRISPR-Cas9 to edit genes was thrust into the spotlight in 2012 when George Church, Jennifer Doudna, Emmanuelle Charpentier, and Feng Zhang harnessed it as a tool to modify targeted regions of genomes.

Is CRISPR a company? ›

CRISPR Therapeutics AG is a Swiss–American biotechnology company headquartered in Zug, Switzerland. In fiscal year 2021, the company had revenues of $915 million, with net income of $378 million.

Is CRISPR natural? ›

CRISPR-Cas9 was adapted from a naturally occurring genome editing system that bacteria use as an immune defense. When infected with viruses, bacteria capture small pieces of the viruses' DNA and insert them into their own DNA in a particular pattern to create segments known as CRISPR arrays.

Does Editas own CRISPR? ›

Editas Medicine's foundational intellectual property includes issued patents covering fundamental aspects of both CRISPR/Cas9 and CRISPR/Cas12a gene editing. Editas Medicine's patents broadly cover CRISPR/Cas9 and CRISPR/Cas12a gene editing in all human cells.

Can you patent Crispr? ›

In the United States, Broad has been allowed or granted 31 CRISPR patents, including 26 patents for CRISPR-Cas9, as well as 3 for CRISPR-Cas12/Cpf1. The USPTO has also granted patents directed to CRISPR-Cas9 to UC Berkeley (UCB), University of Vienna and Emmanuelle Charpentier.

What types of genes are patented? ›

The U.S Supreme Court ruled today that "naturally occurring" human genes cannot be patented because they are a "product of nature," meaning that they cannot be claimed as a human invention. But it also permitted patents based on laboratory reconstructions of human DNA, known as complementary DNAs, or cDNAs.

What was the first gene to be patented? ›

In 1990, the patent office added rules for claiming DNA sequences. Within a year, Amgen patented the first gene, erythropoietin (EPO), used to treat anemia and abused by certain famous athletes.

What are the 4 steps of CRISPR? ›

Several steps to use the CRISPR-CAS9 system for gene editing and genetic engineering are:
  • Select an organism for the experiment.
  • Select a gene of the target location.
  • Select a CRISPR-CAS9 system.
  • Select and Design the sgRNA.
  • Synthesizing and cloning of sgRNA.
  • Delivering the sgRNA and CAS9.
  • Validating the experiment.
3 Aug 2020

How do you deliver CRISPR to cells? ›

CRISPR-Cas9 delivery methods involve both the vehicle (the method of delivery into cells) and cargo (Cas nuclease and guide RNA). CRISPR delivery vehicles fall into three categories: viral, non-viral, and physical. The delivery vehicle will determine whether the Cas nuclease can be delivered as DNA, mRNA, or protein.

How does CRISPR insert genes? ›

The standard form of CRISPR involves adding a protein called Cas9 to a cell along with a piece of guide RNA. The protein searches through the genome until it finds DNA that matches the guide RNA sequence and then cuts the DNA at this point.

Which of the following is the correct order of steps for the CRISPR CAS mechanism? ›

The mechanism of CRISPR/Cas-9 genome editing can be generally divided into three steps: recognition, cleavage, and repair.

What is CRISPR simple explanation? ›

CRISPR is a technology that can be used to edit genes and, as such, will likely change the … world. The essence of CRISPR is simple: it's a way of finding a specific bit of DNA inside a cell. After that, the next step in CRISPR gene editing is usually to alter that piece of DNA.

What is CRISPR and how is it used? ›

A: CRISPR genome editing allows scientists to quickly create cell and animal models, which researchers can use to accelerate research into diseases such as cancer and mental illness. In addition, CRISPR is now being developed as a rapid diagnostic.

How does CRISPR work simplified? ›

The CRISPR arrays allow the bacteria to "remember" the viruses (or closely related ones). If the viruses attack again, the bacteria produce RNA segments from the CRISPR arrays that recognize and attach to specific regions of the viruses' DNA.

Is CRISPR technology nanotechnology? ›

Nanotechnology has greatly contributed to cancer drug delivery. Here, we present the action mechanisms of CRISPR/Cas9, its application in cancer therapy and especially focus on the nanotechnology-based delivery of CRISPR/Cas9 for cancer gene editing and immunotherapy to pave the way for its clinical translation.

Does CRISPR use a viral vector? ›

Lentivirus Vectors

LVs are another type of viral vector that is being used to deliver CRISPR components. It has a higher cloning capacity than the AAV vector.

How do you express on Cas9? ›

The procedure is simple: You mix gRNA and Cas9 with lipid vesicles (liposomes) to form liposome-gRNA/Cas9 complexes. Then you add the complexes directly to your cell cultures. After 24 hours of incubation, the cells will start expressing gRNA and Cas9.

How is gene editing performed? ›

Gene editing is performed using enzymes, particularly nucleases that have been engineered to target a specific DNA sequence, where they introduce cuts into the DNA strands, enabling the removal of existing DNA and the insertion of replacement DNA.

How do you knock out a gene with CRISPR? ›

How CRISPR gene knockout works. A CRISPR-associated (Cas) enzyme is used to cleave target DNA, resulting in a double-strand break (DSB). The Cas enzyme is directed by the guide RNA (gRNA) to a user-defined site in the genome, and then the Cas enzyme cuts the DNA.

When was CRISPR first used for gene editing? ›

CRISPR-Cas9 in the Spotlight

The use of CRISPR-Cas9 to edit genes was thrust into the spotlight in 2012 when George Church, Jennifer Doudna, Emmanuelle Charpentier, and Feng Zhang harnessed it as a tool to modify targeted regions of genomes.

What are the three stages by which CRISPR-Cas immunity occurs and what takes place during each stage? ›

The key steps of CRISPR-Cas immunity. 1) Adaptation: insertion of new spacers into the CRISPR locus. 2) Expression: transcription of the CRISPR locus and processing of CRISPR RNA. 3) Interference: detection and degradation of mobile genetic elements by CRISPR RNA and Cas protein(s).

When using CRISPR to edit a genome What are the mechanisms through which a Cas9 mediated double strand break might be repaired? ›

CRISPR/Cas9-mediated DSB repair mechanism. The CRISPR-associated enzyme Cas9 breaks down the target DNA to create a DSB, the two repeated sequences are further used as templates to produce short crRNAs. Methods for DSB repair include the NHEJ and HDR pathway. The NHEJ pathway creates accurate deletions and insertions.

What are the two components of CRISPR? ›

CRISPR-Cas9 genome editing includes two key components: a single-guide RNA (gRNA) and a CRISPR-associated endonuclease (Cas). The sgRNA and Cas9 are combined into a ribonucleoprotein complex when they are used in CRISPR experiments.

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