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  2. CRISPR gene editing - Wikipedia

    en.wikipedia.org/wiki/CRISPR_gene_editing

    The CRISPR treatment for LCA10 (the most common variant of Leber congenital amaurosis which is the leading cause of inherited childhood blindness) modifies the patient's defective photoreceptor gene. In March 2020, the first patient volunteer in this US-based study, sponsored by Editas Medicine , was given a low-dose of the treatment to test ...

  3. CRISPR interference - Wikipedia

    en.wikipedia.org/wiki/CRISPR_interference

    CRISPR interference (CRISPRi) is a genetic perturbation technique that allows for sequence-specific repression of gene expression in prokaryotic and eukaryotic cells. [1] It was first developed by Stanley Qi and colleagues in the laboratories of Wendell Lim , Adam Arkin, Jonathan Weissman , and Jennifer Doudna . [ 2 ]

  4. Cas12a - Wikipedia

    en.wikipedia.org/wiki/Cas12a

    The CRISPR-Cas12a system consist of a Cas12a enzyme and a guide RNA that finds and positions the complex at the correct spot on the double helix to cleave target DNA. CRISPR-Cas12a systems activity has three stages: [3] Adaptation: Cas1 and Cas2 proteins facilitate the adaptation of small fragments of DNA into the CRISPR array.

  5. Off-target genome editing - Wikipedia

    en.wikipedia.org/wiki/Off-target_genome_editing

    The safety of gene therapy treatment is of utmost concern, especially during clinical trials when off-target modifications can block the further development of a candidate product. [57] Perhaps the most well-known example of modern gene therapy is CAR-T therapy, which is used for the treatment of B-cell lymphoma .

  6. CRISPR-associated transposons - Wikipedia

    en.wikipedia.org/wiki/CRISPR-associated_transposons

    CRISPR-associated transposons or CASTs are mobile genetic elements that have evolved to make use of minimal CRISPR systems for RNA-guided transposition of their DNA. [1] Unlike traditional CRISPR systems that contain interference mechanisms to degrade targeted DNA, CASTs lack proteins and/or protein domains responsible for DNA cleavage. [ 2 ]

  7. FDA panel says Vertex/CRISPR to assess safety risks of ... - AOL

    www.aol.com/news/fda-panel-says-vertex-crispr...

    If the therapy is approved, Vertex has proposed a 15-year follow up of patients to evaluate the safety outcomes of the therapy. A vaso-occlusive crisis occurs when sickled red blood cells block ...

  8. Gene knockout - Wikipedia

    en.wikipedia.org/wiki/Gene_knockout

    CRISPR-based gene knockout is a powerful tool for understanding the genetic basis of disease and for developing new therapies. It is important to note that CRISPR-based gene knockout, like any genetic engineering technique, has the potential to produce unintended or harmful effects on the organism, so it should be used with caution.

  9. Better Buy: CRISPR Therapeutics AG vs. Sangamo ... - AOL

    www.aol.com/news/better-buy-crispr-therapeutics...

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