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  2. CRISPR gene editing - Wikipedia

    en.wikipedia.org/wiki/CRISPR_gene_editing

    CRISPR gene editing (CRISPR, pronounced / ˈkrɪspər / "crisper", refers to " c lustered r egularly i nterspaced s hort p alindromic r epeats") is a genetic engineering technique in molecular biology by which the genomes of living organisms may be modified.

  3. CRISPR - Wikipedia

    en.wikipedia.org/wiki/CRISPR

    CRISPR gene editing is a revolutionary technology that allows for precise, targeted modifications to the DNA of living organisms. Developed from a natural defense mechanism found in bacteria, CRISPR-Cas9 is the most commonly used system, that allows "cutting" of DNA at specific locations and either delete, modify, or insert genetic material.

  4. CRISPR activation - Wikipedia

    en.wikipedia.org/wiki/CRISPR_activation

    CRISPR activation (CRISPRa) is a gene regulation technique that utilizes an engineered form of the CRISPR-Cas9 system to enhance the expression of specific genes without altering the underlying DNA sequence. Unlike traditional CRISPR-Cas9, which introduces double-strand breaks to edit genes, CRISPRa employs a modified, catalytically inactive ...

  5. Gene therapy - Wikipedia

    en.wikipedia.org/wiki/Gene_therapy

    Gene therapy is a medical technology that aims to produce a therapeutic effect through the manipulation of gene expression or through altering the biological properties of living cells. [ 1 ][ 2 ][ 3 ] The first attempt at modifying human DNA was performed in 1980, by Martin Cline, but the first successful nuclear gene transfer in humans ...

  6. FDA approves cure for sickle cell disease, the first ... - AOL

    www.aol.com/news/fda-approves-cure-sickle-cell...

    December 8, 2023 at 1:19 PM. The Food and Drug Administration on Friday approved a powerful treatment for sickle cell disease, a devastating illness that affects more than 100,000 Americans, the ...

  7. Genome-wide CRISPR-Cas9 knockout screens - Wikipedia

    en.wikipedia.org/wiki/Genome-wide_CRISPR-Cas9...

    The exact protocol for lentiviral production will vary depending on the research aim and applied library. [35] [43] [44] If a two vector-system is used, for example, cells are sequentially transduced with Cas9 and sgRNA in a two-step procedure. [35] [44] Although more complex, this has the advantage of a higher titre for the sgRNA library virus ...

  8. Cas12a - Wikipedia

    en.wikipedia.org/wiki/Cas12a

    Cas12a (C RISPR- as sociated protein 12a, previously known as Cpf1) is a subtype of Cas12 proteins and an RNA-guided endonuclease that forms part of the CRISPR system in some bacteria and archaea. In CRISPR systems, Cas12a serves to destroy the genetic material of viruses and other foreign DNA, thereby protecting the cell from infection.

  9. CRISPR Therapeutics - Wikipedia

    en.wikipedia.org/wiki/CRISPR_Therapeutics

    CRISPR Therapeutics AG is a Swiss–American biotechnology company headquartered in Zug, Switzerland. It was one of the first companies formed to utilize the CRISPR gene editing platform to develop medicines for the treatment of various rare and common diseases. [ 2 ][ 3 ] The company has approximately 500 employees and has offices in Zug ...