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Article

Molecular characterization of precise in vivo targeted gene integration in human cells using AAVHSC15.

PloS one - 1 Jan 2020

Chen Huei-Mei, Resendes Rachel, Ghodssi Azita, Sookiasian Danielle, Tian Michael, Dollive Serena, Adamson-Small Laura, Avila Nancy, Tazearslan Cagdas, Thompson John F, Ellsworth Jeff L, Francone Omar, Seymour Albert, Wright Jason B

Abstract excerpt

Targeted gene integration via precise homologous recombination (HR)-based gene editing has the potential to correct genetic diseases. AAV (adeno-associated virus) can mediate nuclease-free gene integration at a disease-causing locus. Therapeutic application of AAV gene integration requires quantitative molecular characterization of the edited sequence that overcome technical obstacles such as excess episomal...

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PMID: 32453743
DOI: 10.1371/journal.pone.0233373
PMCID: PMC7250422

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PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 6 Sept 2026. Manifest publication-graph-cohort-2026-09-06.1.

Source receipt: pubmed, retrieved 6 Sept 2026.

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