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:	Zeenath Unnisa, John K. Yoon, Jeffrey W. Schindler, Chris Mason, Niek P. van Til
格式:	Artigo
語言:	Inglês
出版:	MDPI AG 2022-01-01
叢編:	Biomedicines
主題:	Pompe disease enzyme replacement therapy adeno-associated viral vector hematopoietic stem cell lentiviral vectors
在線閱讀:	https://www.mdpi.com/2227-9059/10/2/302
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