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Article

Prime Editing for Human Gene Therapy: Where Are We Now?

Cells - 7 Feb 2023

Godbout Kelly, Tremblay Jacques P

Abstract excerpt

Gene therapy holds tremendous potential in the treatment of inherited diseases. Unlike traditional medicines, which only treat the symptoms, gene therapy has the potential to cure the disease by addressing the root of the problem: genetic mutations. The discovery of CRISPR/Cas9 in 2012 paved the way for the development of those therapies. Improvement of this system led to the recent development of an outstanding...

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PMID: 36831203
DOI: 10.3390/cells12040536
PMCID: PMC9954691

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