Therapeutic advantages of combined gene/cell therapy strategies in a murine model of GM2 gangliosidosis

Therapeutic advantages of combined gene/cell therapy strategies in a murine model of GM2 gangliosidosis

Genetic deficiency of β-N-acetylhexosaminidase (Hex) functionality leads to accumulation of GM2 ganglioside in Tay-Sachs disease and Sandhoff disease (SD), which presently lack approved therapies. Current experimental gene therapy (GT) approaches with adeno-associated viral vectors (AAVs) still pose...

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Bibliographic Details
Main Authors: Davide Sala, Francesca Ornaghi, Francesco Morena, Chiara Argentati, Manuela Valsecchi, Valeria Alberizzi, Roberta Di Guardo, Alessandra Bolino, Massimo Aureli, Sabata Martino, Angela Gritti
Format: Article
Language:English
Published: Elsevier 2022-06-01
Series:Molecular Therapy: Methods & Clinical Development
Subjects:
lysosomal storage disorders
GM2 gangliosidosis
gene therapy
CNS
bone marrow transplantation
lentiviral vectors
Online Access:http://www.sciencedirect.com/science/article/pii/S2329050122000419

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