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Screening chimeric GAA variants in preclinical study results in hematopoietic stem cell gene therapy candidate vectors for Pompe disease

Pompe disease is a rare genetic neuromuscular disorder caused by acid α-glucosidase (GAA) deficiency resulting in lysosomal glycogen accumulation and progressive myopathy. Enzyme replacement therapy, the current standard of care, penetrates poorly into the skeletal muscles and the peripheral and cen...

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Detalles Bibliográficos
Autores principales:	Dogan, Yildirim, Barese, Cecilia N., Schindler, Jeffrey W., Yoon, John K., Unnisa, Zeenath, Guda, Swaroopa, Jacobs, Mary E., Oborski, Christine, Maiwald, Tim, Clarke, Diana L., Schambach, Axel, Pfeifer, Richard, Harper, Claudia, Mason, Chris, van Til, Niek P.
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/PMC9676529/ https://www.ncbi.nlm.nih.gov/pubmed/36419467 http://dx.doi.org/10.1016/j.omtm.2022.10.017

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