IGF2-tagging of GAA promotes full correction of murine Pompe disease at a clinically relevant dosage of lentiviral gene therapy

Pompe disease is caused by deficiency of acid α-glucosidase (GAA), resulting in glycogen accumulation in various tissues, including cardiac and skeletal muscles and the central nervous system (CNS). Enzyme replacement therapy (ERT) improves cardiac, motor, and respiratory functions but is limited by...

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Detalles Bibliográficos
Autores principales: Liang, Qiushi, Catalano, Fabio, Vlaar, Eva C., Pijnenburg, Joon M., Stok, Merel, van Helsdingen, Yvette, Vulto, Arnold G., van der Ploeg, Ans T., van Til, Niek P., Pijnappel, W.W.M. Pim
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/PMC9573825/
https://www.ncbi.nlm.nih.gov/pubmed/36284764
http://dx.doi.org/10.1016/j.omtm.2022.09.010

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