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

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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Bibliographic Details
Main Authors: Marine Geoffroy, Louna Pili, Valentina Buffa, Maëlle Caroff, Anne Bigot, Evelyne Gicquel, Grégory Rouby, Isabelle Richard, Romain Fragnoud
Format: Article
Language:English
Published: MDPI AG 2023-10-01
Series:Cells
Subjects:
Limb-Girdle Muscular Dystrophy Type 9
Fukutin-related protein (FKRP)
CRISPR
knock-out
potency assay
gene therapy
Online Access:https://www.mdpi.com/2073-4409/12/20/2444

Internet

https://www.mdpi.com/2073-4409/12/20/2444

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