Targeting the Apoa1 locus for liver-directed gene therapy

Targeting the Apoa1 locus for liver-directed gene therapy

Clinical application of somatic genome editing requires therapeutics that are generalizable to a broad range of patients. Targeted insertion of promoterless transgenes can ensure that edits are permanent and broadly applicable while minimizing risks of off-target integration. In the liver, the Album...

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Bibliographic Details
Main Authors: Marco De Giorgi, Ang Li, Ayrea Hurley, Mercedes Barzi, Alexandria M. Doerfler, Nikitha A. Cherayil, Harrison E. Smith, Jonathan D. Brown, Charles Y. Lin, Karl-Dimiter Bissig, Gang Bao, William R. Lagor
Format: Article
Language:English
Published: Elsevier 2021-06-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
AAV
CRISPR-Cas9
gene therapy
genome editing
gene targeting
Apoa1
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050121000772

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http://www.sciencedirect.com/science/article/pii/S2329050121000772

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