therapy, can deliver a functional copy of the acid alpha-glucosidase (GAA) gene directly in muscle cells in adults with late-onset Pompe disease. GAA is responsible for metabolising glycogen ...

Astellas has licensed rights to a drug developed by Selecta Biosciences that could make more patients eligible for treatment with its gene ... therapy for inherited neuromuscular disorder Pompe ...

and Pompe disease. AskBio’s gene therapy platform includes Pro10™, an industry-leading proprietary cell line manufacturing process, and an extensive array of capsids and promoters. With global ...

Deficiency of the pancreatic progenitor cell differentiation and proliferation factor gene was identified as a risk factor ...

“We have been dreaming of gene therapy as a treatment for this disease,” he said. Government Science Data: Vast quantities of climate and environmental information have been removed from ...