Mutation-Independent Allele-Specific Editing by CRISPR-Cas9, a Novel Approach to Treat Autosomal Dominant Disease-Reference-Cited by-同舟云学术

Mutation-Independent Allele-Specific Editing by CRISPR-Cas9, a Novel Approach to Treat Autosomal Dominant Disease

Published:2020-08 Issue:8 Volume:28 Page:1846-1857
ISSN:1525-0016
Container-title:Molecular Therapy
language:en
Short-container-title:Molecular Therapy

Author:

Christie Kathleen A.,Robertson Louise J.,Conway Caroline,Blighe Kevin,DeDionisio Larry A.,Chao-Shern Connie,Kowalczyk Amanda M.,Marshall John,Turnbull Doug,Nesbit M. Andrew,Moore C.B. Tara

Funder

Avellino Labs USA

Publisher

Elsevier BV

Subject

Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine

Reference59 articles.

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4. Dissecting disease inheritance modes in a three-dimensional protein network challenges the “guilt-by-association” principle;Guo;Am. J. Hum. Genet.,2013

5. Treatment of autosomal dominant hearing loss by in vivo delivery of genome editing agents;Gao;Nature,2018

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3. Allele-specific CRISPR/Cas9 editing inactivates a single nucleotide variant associated with collagen VI muscular dystrophy;2024-03-22

4. Epigenetic editing for autosomal dominant neurological disorders;Frontiers in Genome Editing;2024-03-06

5. Functional benefit of CRISPR/Cas9-induced allele deletion forRYR1dominant mutation;2024-01-25