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Article

Full-length dystrophin restoration via targeted exon integration by AAV-CRISPR in a humanized mouse model of Duchenne muscular dystrophy.

Molecular therapy : the journal of the American Society of Gene Therapy - 3 Nov 2021

Pickar-Oliver Adrian, Gough Veronica, Bohning Joel D, Liu Siyan, Robinson-Hamm Jacqueline N, Daniels Heather, Majoros William H, Devlin Garth, Asokan Aravind, Gersbach Charles A

Abstract excerpt

Targeted gene-editing strategies have emerged as promising therapeutic approaches for the permanent treatment of inherited genetic diseases. However, precise gene correction and insertion approaches using homology-directed repair are still limited by low efficiencies. Consequently, many gene-editing strategies have focused on removal or disruption, rather than repair, of genomic DNA. In contrast,...

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Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

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Source

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Identifiers

PMID: 34509668
DOI: 10.1016/j.ymthe.2021.09.003
PMCID: PMC8571168

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 7 Sept 2026. Manifest publication-graph-cohort-2026-09-07.1.

Source receipt: pubmed, retrieved 7 Sept 2026.

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