Improvements of nuclease and nickase gene modi cation techniques for the treatment of genetic diseases

HIGHLIGHTS

• who: ZFN and colleagues from the Graphite Bio, Inc, United States have published the article: Improvements of nuclease and nickase gene modi cation techniques for the treatment of genetic diseases, in the Journal: (JOURNAL)
• what: This approach has also been used in pig model of autosomal dominant Retinitis Pigmentosa (adRP) for the correction of P23H mutation in the rhodopsin (RHO) gene (Jalligampala et_al, 2021) and in non-human primates for the modification of the proprotein convertase subtilisin/kexin type 9 (PCSK9) gene responsible for hypercholesterolemia (Wang et_al, 2018a; Wang et_al, 2021). homologous allelic sites (Epinat . . .
  

   

  

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