MEDizzy
MEDizzy
MUNENDRA A
MUNENDRA Aalmost 7 years ago
Injection of novel gene therapy vector prolonged survival in mouse model of Pompe disease

Injection of novel gene therapy vector prolonged survival in mouse model of Pompe disease

A new study has shown that a single injection of a novel adeno-associated vector (AAV)-based therapy can result in improved enzyme activ-ity and glycogen clearance as well as prolonged survival in a mouse model of Pompe dis-ease. Young mice received an injection of AAVB1-GAA gene therapy, which delivers a normal copy of the gene for the lysosomal enzyme alpha glucosidase (GAA), which is mutated in Pompe disease, a lysosoma disease. The positive effects of gene replacement seen in adult mice are described in an article published in Human Gene Therapy.

Source: https://medicalxpress.com/news/2018-08-gene-therapy-vector-prolonged-survival.html
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Top rated comment
almost 7 years ago

Type2 glycogen storage disease??

almost 7 years ago

Generally besttreatmentnow

almost 7 years ago

Generally best treatment now

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almost 7 years ago

Generallybesttreatmentnow

almost 7 years ago

It's defect in storage of glycogen

almost 7 years ago

This fact S,,,,,

almost 7 years ago

Whether any attempt s made for germ cell gene therapy

almost 7 years ago

🤔

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