Gene Therapy Developments for Pompe Disease

Pompe disease is an inherited neuromuscular disorder caused by deficiency of the lysosomal enzyme acid alpha-glucosidase (GAA). The most severe form is infantile-onset Pompe disease, presenting shortly after birth with symptoms of cardiomyopathy, respiratory failure and skeletal muscle weakness. Lat...

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Detalles Bibliográficos
Autores principales: Unnisa, Zeenath, Yoon, John K., Schindler, Jeffrey W., Mason, Chris, van Til, Niek P.
Formato: Online Artículo Texto
Lenguaje:English
Publicado: MDPI 2022
Materias:
Review
Acceso en línea:https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8869611/
https://www.ncbi.nlm.nih.gov/pubmed/35203513
http://dx.doi.org/10.3390/biomedicines10020302

Internet

https://www.ncbi.nlm.nih.gov/pmc/articles/PMC8869611/
https://www.ncbi.nlm.nih.gov/pubmed/35203513
http://dx.doi.org/10.3390/biomedicines10020302

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