Cargando…
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...
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 |
Ejemplares similares
-
A Self-Deleting AAV-CRISPR System for In Vivo Genome Editing
por: Li, Ang, et al.
Publicado: (2018) -
AAV-CRISPR Gene Editing Is Negated by Pre-existing Immunity to Cas9
por: Li, Ang, et al.
Publicado: (2020) -
Treating Cardiovascular Disease with Liver Genome Engineering
por: Hurley, Ayrea, et al.
Publicado: (2022) -
In vivo genome editing at the albumin locus to treat methylmalonic acidemia
por: Schneller, Jessica L., et al.
Publicado: (2021) -
LPA disruption with AAV-CRISPR potently lowers plasma apo(a) in transgenic mouse model: A proof-of-concept study
por: Doerfler, Alexandria M., et al.
Publicado: (2022)