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...

Celý popis

Podrobná bibliografie

Hlavní autoři:	Davide Sala, Francesca Ornaghi, Francesco Morena, Chiara Argentati, Manuela Valsecchi, Valeria Alberizzi, Roberta Di Guardo, Alessandra Bolino, Massimo Aureli, Sabata Martino, Angela Gritti
Médium:	Artigo
Jazyk:	Inglês
Vydáno:	Elsevier 2022-06-01
Edice:	Molecular Therapy: Methods & Clinical Development
Témata:	lysosomal storage disorders GM2 gangliosidosis gene therapy CNS bone marrow transplantation lentiviral vectors
On-line přístup:	http://www.sciencedirect.com/science/article/pii/S2329050122000419
Tagy:	Přidat tag Žádné tagy, Buďte první, kdo vytvoří štítek k tomuto záznamu!