Expanding the repertoire of CRISPR-based genomic editors

Simon Ng — Thu, 04 Nov 2021 18:04:42 +0000

Devyn Smith, PhD, Arbor Biotechnologies, Cambridge, MA, provides a brief overview of the tailored nuclease therapies developed at Arbor Biotechnologies. Multiple nucleases have been discovered that are superior to the original Cas9 endonuclease and a portfolio of nucleases, which have been modified to improve efficacy, has been developed. Certain nucleases can be adapted for specific diseases such as Huntington’s disease, a trinucleotide repeat disorder, by having the optimal protospacer adjacent motif (PAM). This interview took place at Meeting on the Mesa 2021.

Huntington’s Disease – VJRegenMed

Expanding the repertoire of CRISPR-based genomic editors