CRISPR

Extracted Attributes

• Type

  DNA sequences, Gene-editing technology

• Found In

  Prokaryotic organisms (bacteria, archaea)

• Full Name

  Clustered Regularly Interspaced Short Palindromic Repeats

• Applications

  Basic biological research, biotechnology, disease treatment, creation of genetically modified organisms, control of pathogens and pests, epigenome editing

• Characteristics

  Precise, cost-effective, efficient

• Medical Applications

  Treatment of inherited genetic diseases, diseases from somatic mutations (e.g., cancer), neurodegenerative diseases, blood disorders, ocular disorders

• Primary Function (Natural)

  Antiviral defense system, acquired immunity against bacteriophages

• Key Components (Technology)

  CRISPR sequences, Cas9 enzyme, guide RNA, Base Editors

Timeline

• CRISPR system discovered in microbes. (Source: Web Search (Stanford Report))

  1987-XX-XX

• Researchers discovered CRISPR functions as an immune system in microbes. (Source: Web Search (Stanford Report))

  2005-XX-XX

• First trial of a CRISPR cell therapy performed, treating patients with sickle cell disease. (Source: Web Search (Synthego))

  2019-XX-XX

• Emmanuelle Charpentier and Jennifer Doudna were awarded the Nobel Prize in Chemistry for the development of the CRISPR-Cas9 genome editing technique. (Source: Summary, Wikipedia, DBPedia)

  2020-XX-XX

• The PTAB favored Feng Zhang for an invention covering the application of CRISPR-Cas9 in eukaryotic cells. (Source: Summary, DBPedia)

  2022-XX-XX

• CRISPR technology, specifically using Base Editors, was successfully employed by physician Rebecca Arens Nicholas and UCSF researchers to treat a fatal genetic disease in an infant. (Source: Related Documents)

  XXXX-XX-XX

CRISPR

CRISPR (; acronym of clustered regularly interspaced short palindromic repeats) is a family of DNA sequences found in the genomes of prokaryotic organisms such as bacteria and archaea. Each sequence within an individual prokaryotic CRISPR is derived from a DNA fragment of a bacteriophage that had previously infected the prokaryote or one of its ancestors. These sequences are used to detect and destroy DNA from similar bacteriophages during subsequent infections. Hence these sequences play a key role in the antiviral (i.e. anti-phage) defense system of prokaryotes and provide a form of heritable, acquired immunity. CRISPR is found in approximately 50% of sequenced bacterial genomes and nearly 90% of sequenced archaea. Cas9 (or "CRISPR-associated protein 9") is an enzyme that uses CRISPR sequences as a guide to recognize and open up specific strands of DNA that are complementary to the CRISPR sequence. Cas9 enzymes together with CRISPR sequences form the basis of a technology known as CRISPR-Cas9 that can be used to edit genes within living organisms. This editing process has a wide variety of applications including basic biological research, development of biotechnological products, and treatment of diseases. The development of the CRISPR-Cas9 genome editing technique was recognized by the Nobel Prize in Chemistry in 2020 awarded to Emmanuelle Charpentier and Jennifer Doudna.

Web Search Results

• CRISPR gene editing - Wikipedia

  The clustered regularly interspaced short palindromic repeats (CRISPR)/Cas9 system is a gene-editing technology that can induce double-strand breaks (DSBs) anywhere guide ribonucleic acids (gRNA) can bind with the protospacer adjacent motif (PAM) sequence.( Single-strand nicks can also be induced by Cas9 active-site mutants,( also known as Cas9 nickases.( By simply changing the sequence of gRNA, the Cas9-endonuclease can be delivered to a gene of interest and induce DSBs.( The efficiency of [...] CRISPR simplifies the creation of genetically modified organisms for research which mimic disease or show what happens when a gene is knocked down or mutated. CRISPR may be used at the germline level to create organisms in which the targeted gene is changed everywhere (i.e. in all cells/tissues/organs of a multicellular organism), or it may be used in non-germline cells to create local changes that only affect certain cell populations within the organism.( [...] The technique is considered highly significant in biotechnology and medicine as it enables editing genomes _in vivo_ and is precise, cost-effective, and efficient. It can be used in the creation of new medicines, agricultural products, and genetically modified organisms, or as a means of controlling pathogens and pests. It also offers potential in the treatment of inherited genetic diseases as well as diseases arising from somatic mutations such as cancer. However, its use in human germline

• Questions and Answers about CRISPR | Broad Institute

  A: “CRISPR” (pronounced “crisper”) stands for Clustered Regularly Interspaced Short Palindromic Repeats, which are the hallmark of a bacterial defense system that forms the basis for CRISPR-Cas9 genome editing technology. In the field of genome engineering, the term “CRISPR” or “CRISPR-Cas9” is often used loosely to refer to the various CRISPR-Cas9 and -CPF1, (and other) systems that can be programmed to target specific stretches of genetic code and to edit DNA at precise locations, as well as

• Applications of CRISPR technologies to the development of gene ...

  The Clustered Regularly Interspaced Short Palindromic Repeats (CRISPR) are originally discovered as bacterial immune systems and were subsequently investigated as versatile tools for genome editing (1-3). Generally, CRISPR system consists of a combination of single or multiple protein components with nucleic acid-cutting functions and a guide RNA that determines the target nucleic acids. In the cases of DNA targeting CRISPRs, double-strand DNA breaks (DSB) occur at the target loci with a [...] Gene editing technologies have developed through various generations. The first generation included zinc-finger nucleases (ZFN), while the second generation comprised transcription activator-like effector nucleases (TALEN). The discovery and advancement of third generation CRISPR brought rapid progress in the gene editing technology (21-24). The CRISPR system is a form of adaptive immunity that allows bacteria and archaea to respond to exogenous viral invasions (5). In bacteria, the CRISPR [...] repair process of the cleaved DNA induces genome editing at the site of DNA breaks. The results of CRISPR genome editing include insertions or deletions of variable sequences at the target site or insert externally delivered DNA sequences. In eukaryotic cells, the repair of DSB involves three processes: NHEJ, homologous recombination (HR), and MMEJ (39-41). These DNA repair processes function in a mutually exclusive or complementary manner, depending on the in vivo and cellular context (39, 42,

• What is CRISPR? A bioengineer explains - Stanford Report

  Qi: CRISPR stands for “clustered interspaced short palindromic repeats.” Biologists use the term to describe the “genetic appearance” of a system that was discovered in microbes – including bacteria and archaea – as early as 1987. For a long time, no one really understood what it did, but around 2005, researchers discovered CRISPR is an immune system. It’s used by microbes to help protect themselves from invading viruses. To stop the invaders, the microbes use CRISPR to recognize and eliminate [...] Back to the list of questions `5. Why is it such a big deal?` The short answer: CRISPR can precisely modify a piece of DNA or its chemistry (so-called epigenetics) in the human body, making it a potential tool for clinical uses in the biomedical sciences. Qi:CRISPR is a molecule and tool desired by everyone who works in the life sciences, biomedical research, and clinical settings. Its high precision is unparalleled and enables many uses including gene therapy. [...] Over the past decade, CRISPR has taken the biomedical world and life sciences by storm for its ability to easily and precisely edit DNA. Here, Stanford University bioengineer Stanley Qi explains how CRISPR works, why it’s such an important tool, and how it could be used in the future – including current developments in using CRISPR to edit the epigenome, which involves altering the chemistry of DNA instead of the DNA sequence itself.

• What is CRISPR: Your Ultimate Guide | Synthego

  CRISPR 101 eBook Everything You Need to Know. What Is CRISPR & Why Is It Important? ------------------------------------- CRISPR stands for Clustered Regularly Interspaced Short Palindromic Repeats. It is a component of bacterial immune systems that can cut DNA, and has been repurposed as a gene editing tool. It acts as a precise pair of molecular scissors that can cut a target DNA sequence, directed by a customizable guide. [...] The CRISPR system is the basis of adaptive immunity in bacteria and archaea. It utilizes Cas nucleases, which are enzymes that can bind and create double-stranded breaks (DSBs) in DNA. When a bacterium is infected by a virus, it uses a Cas nuclease to snip off a piece of viral DNA known as a protospacer. This fragment is stored in the bacterial genome with fragments from other viruses that have previously infected the cell - an immune memory. These viral spacer fragments are placed between [...] CRISPR is poised to revolutionize medicine, with the potential to cure a range of genetic diseases, including neurodegenerative disease, blood disorders, cancer, and ocular disorders. As we mentioned earlier, the first trial of a CRISPR cell therapy was performed in 2019, treating patients with sickle cell disease. The treatment restored fetal hemoglobin, eliminating the need for a functional copy of adult hemoglobin.

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CRISPR (/ˈkrɪspər/) (an acronym for clustered regularly interspaced short palindromic repeats) is a family of DNA sequences found in the genomes of prokaryotic organisms such as bacteria and archaea. These sequences are derived from DNA fragments of bacteriophages that had previously infected the prokaryote. They are used to detect and destroy DNA from similar bacteriophages during subsequent infections. Hence these sequences play a key role in the antiviral (i.e. anti-phage) defense system of prokaryotes and provide a form of acquired immunity. CRISPR is found in approximately 50% of sequenced bacterial genomes and nearly 90% of sequenced archaea. Cas9 (or "CRISPR-associated protein 9") is an enzyme that uses CRISPR sequences as a guide to recognize and cleave specific strands of DNA that are complementary to the CRISPR sequence. Cas9 enzymes together with CRISPR sequences form the basis of a technology known as CRISPR-Cas9 that can be used to edit genes within organisms. This editing process has a wide variety of applications including basic biological research, development of biotechnological products, and treatment of diseases. The development of the CRISPR-Cas9 genome editing technique was recognized by the Nobel Prize in Chemistry in 2020 which was awarded to Emmanuelle Charpentier and Jennifer Doudna. In 2022, based on evidence presented in Interference 106, 115, the PTAB tipped the scale for invention covering application of CRISPR-Cas9 in eukaryotic cells in favour of Feng Zhang, a professor of the Broad Institute.