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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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Detalles Bibliográficos
Autores principales: De Giorgi, Marco, Li, Ang, Hurley, Ayrea, Barzi, Mercedes, Doerfler, Alexandria M., Cherayil, Nikitha A., Smith, Harrison E., Brown, Jonathan D., Lin, Charles Y., Bissig, Karl-Dimiter, Bao, Gang, Lagor, William R.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2021
Materias:
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8166646/
https://www.ncbi.nlm.nih.gov/pubmed/34141821
http://dx.doi.org/10.1016/j.omtm.2021.04.011