Design and validation of a GMP stem cell manufacturing protocol for MPSII hematopoietic stem cell gene therapy

Design and validation of a GMP stem cell manufacturing protocol for MPSII hematopoietic stem cell gene therapy

Hematopoietic stem cell gene therapy (HSCGT) is a promising therapeutic strategy for the treatment of neurodegenerative, metabolic disorders. The approach involves the ex vivo introduction of a missing gene into patients’ own stem cells via lentiviral-mediated transduction (TD). Once transplanted ba...

Full description

Bibliographic Details
Main Authors: Stuart Ellison, Karen Buckland, Yuko Learmonth, Victoria Day, Spandan Kalra, Lauren Howe, Francisco José Roman-Rodriguez, Jose Bonafont, Laura Booth, Rebecca Holley, Jon Smythe, Simon Jones, Adrian Thrasher, Claire Booth, Brian W. Bigger
Format: Article
Language:English
Published: Elsevier 2024-06-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
hematopoietic stem cell gene therapy
MPSII
mucopolysaccharidosis
GMP cell manufacturing
transduction enhancers
cleanroom validation studies
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050124000871

Internet

http://www.sciencedirect.com/science/article/pii/S2329050124000871

Similar Items