Durable HTT silencing using non-evolved dCas9 epigenome editors in patient-derived cells

Durable HTT silencing using non-evolved dCas9 epigenome editors in patient-derived cells

Huntington’s disease (HD) is an autosomal dominant neurodegenerative disorder caused by a trinucleotide repeat expansion in exon 1 of the huntingtin (HTT) gene. Nuclease-deficient Cas9 protein (dCas9) epigenetic editing for targeted gene regulation is a promising therapeutic approach for HD through...

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Bibliographic Details
Main Authors: Jennifer J. Waldo, Julian A.N.M. Halmai, Ankita Singh, Casiana E. Gonzalez, Yi-An Chen, Shaylyn A. Carthen, Jan A. Nolta, Kyle D. Fink
Format: Article
Language:English
Published: Elsevier 2025-06-01
Series:Molecular Therapy: Nucleic Acids
Subjects:
MT: RNA/DNA Editing
CRISPR
epigenetics
dCas9
DNA methylation
Huntington’s disease
Online Access:http://www.sciencedirect.com/science/article/pii/S2162253125001155
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