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In February 2020, a US trial showed safe CRISPR gene editing on three cancer patients. [38] In October 2020, researchers Emmanuelle Charpentier and Jennifer Doudna were awarded the Nobel Prize in Chemistry for their work in this field. [39] [40] They made history as the first two women to share this award without a male contributor. [41] [5]
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.
The "iap" gene in gut microbe E. coli was sequenced by Ishino and his colleagues in 1987. [6] As the DNA segment used was longer than the gene itself, they accidentally discovered a partial DNA sequence of then-unnamed CRISPR in the process, [7] which would eventually become the basis of CRISPR gene editing.
Genome editing, or genome engineering, or gene editing, is a type of genetic engineering in which DNA is inserted, deleted, modified or replaced in the genome of a living organism. Unlike early genetic engineering techniques that randomly inserts genetic material into a host genome, genome editing targets the insertions to site-specific locations.
It is far less effective at gene correction. Methods of base editing are under development in which a “nuclease-dead” Cas 9 endonuclease or a related enzyme is used for gene targeting while a linked deaminase enzyme makes a targeted base change in the DNA. [69] The most recent refinement of CRISPR-Cas9 is called Prime Editing.
In June 2021, Intellia made history by announcing interim Phase 1 data for NTLA-2001, demonstrating the ability to precisely edit target cells within the body to treat genetic disease with a single intravenous infusion of CRISPR. This was the first time any human clinical data has been published for an in vivo gene editing therapeutic candidate.
Gene editing is used for a variety of tasks including the modifying of crops, the modifying of bacteria, and the modifying of disease-causing genetic mutations in patients. When only a single edited cell line is required, CRISPR/Cas combined with the endogenous DNA repair efficiency is sufficient to obtain an edited cell line.
CRISPR gene editing based on Clustered regularly interspaced short palindromic repeats (CRISPR) -Cas9 is an enzyme that uses the gene sequences [7] to help control, cleave, and separate specific DNA sequences that are complementary to a CRISPR sequence. [8] [9] These sequences and enzymes were originally derived from bacteriophages. [10]
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