Approaches to purification and concentration of rAAV vectors for gene therapy
Sign Up to like & getrecommendations! Published in 2022 at "BioEssays"
DOI: 10.1002/bies.202200019
Abstract: Recombinant adeno‐associated viruses (rAAVs) are promising vectors for the delivery of various genetic constructs into eukaryotic cells. rAAVs have a number of properties that make it possible to successfully use them both in vitro and… read more here.
Keywords: purification; raav vectors; concentration raav; purification concentration ... See more keywords
Continued analysis of GAA mice treated with novel hybrid promoter rAAV vectors expressing acid alpha-glucosidase
Sign Up to like & getrecommendations! Published in 2019 at "Molecular Genetics and Metabolism"
DOI: 10.1016/j.ymgme.2018.12.168
Abstract: Pompe disease is caused by mutations in the acid alpha-glucosidase gene (GAA) that is responsible for processing lysosomal glycogen. Patients with Pompe disease exhibit clinical phenotypes across a variety of tissues, including glycogen buildup in… read more here.
Keywords: mice; muscle; gaa; raav vectors ... See more keywords