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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Detalles Bibliográficos
Autores principales: Sala, Davide, Ornaghi, Francesca, Morena, Francesco, Argentati, Chiara, Valsecchi, Manuela, Alberizzi, Valeria, Di Guardo, Roberta, Bolino, Alessandra, Aureli, Massimo, Martino, Sabata, Gritti, Angela
Formato: Online Artículo Texto
Lenguaje:English
Publicado: American Society of Gene & Cell Therapy 2022
Materias:
Original Article
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8983315/
https://www.ncbi.nlm.nih.gov/pubmed/35434178
http://dx.doi.org/10.1016/j.omtm.2022.03.011

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