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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| Principais autores: | , , , , |
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| Formato: | Artigo |
| Idioma: | Inglês |
| Publicado: |
MDPI AG
2022-01-01
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| Series: | Biomedicines |
| Assuntos: | |
| Acceso en liña: | https://www.mdpi.com/2227-9059/10/2/302 |
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