CRISPR-Cas9 KO Cell Line Generation and Development of a Cell-Based Potency Assay for rAAV-FKRP Gene Therapy

Limb-Girdle Muscular Dystrophy R9 (LGMDR9) is a dystroglycanopathy caused by Fukutin-related protein (FKRP) defects leading to the deficiency of α-DG glycosylation, essential to membrane integrity. Recombinant adeno-associated viral vector (rAAV) gene therapy offers great therapeutic promise for suc...

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Detalles Bibliográficos
Autores principales: Geoffroy, Marine, Pili, Louna, Buffa, Valentina, Caroff, Maëlle, Bigot, Anne, Gicquel, Evelyne, Rouby, Grégory, Richard, Isabelle, Fragnoud, Romain
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2023
Materias:
Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10604961/
https://www.ncbi.nlm.nih.gov/pubmed/37887288
http://dx.doi.org/10.3390/cells12202444

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC10604961/
https://www.ncbi.nlm.nih.gov/pubmed/37887288
http://dx.doi.org/10.3390/cells12202444

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