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On 26 November 2018, The CRISPR Journal published ahead of print an article by He, Ryan Ferrell, Chen Yuanlin, Qin Jinzhou, and Chen Yangran in which the authors justified the ethical use of CRISPR gene editing in humans. [74] As the news of CRISPR babies broke out, the editors reexamined the paper and retracted it on 28 December, announcing:
Off-target genome editing refers to nonspecific and unintended genetic modifications that can arise through the use of engineered nuclease technologies such as: clustered, regularly interspaced, short palindromic repeats ()-Cas9, transcription activator-like effector nucleases (), meganucleases, and zinc finger nucleases (ZFN). [1]
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.
CRISPR gene editing (CRISPR, pronounced / ˈ k r ɪ s p ə r / (crisper), refers to a clustered regularly interspaced short palindromic repeats") is a genetic engineering technique in molecular biology by which the genomes of living organisms may be modified.
The CRISPR/Cas (clustered regularly interspaced short palindromic repeats/CRISPR associated nucleases) system was originally discovered to be an acquired immune response mechanism used by archaea and bacteria. It has since been adopted for use as a tool in the genetic engineering of higher organisms.
See: Guide RNA, CRISPR. Complementary base pairing between the sgRNA and genomic DNA allows targeting of Cas9 or dCas9. A small guide RNA (sgRNA), or gRNA is an RNA with around 20 nucleotides used to direct Cas9 or dCas9 to their targets. gRNAs contain two major regions of importance for CRISPR systems: the scaffold and spacer regions.
The different generations of nucleases used for genome editing and the DNA repair pathways used to modify target DNA. 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.
CRISPR Therapeutics is applying this technology platform to research, develop and commercialize medicines for various diseases including sickle cell disease, beta thalassemia, various cancers, type 1 diabetes, and cardiovascular diseases. [7] The CEO is Samarth Kulkarni, PhD, who joined the company in 2015 as chief business officer.