Assessment of adeno-associated virus gene therapies efficacy on acid alpha-glucosidase restoration and glycogen storage correction in cardiac muscle of Pompe disease mice using synchrotron infrared and ultraviolet microspectroscopies

Assessment of adeno-associated virus gene therapies efficacy on acid alpha-glucosidase restoration and glycogen storage correction in cardiac muscle of Pompe disease mice using synchrotron infrared and ultraviolet microspectroscopies

Pompe disease (glycogen storage disease type II) is a lysosomal storage disorder due to a mutation in the gene that encodes acid alpha-glucosidase (GAA). GAA deficiency causes the excessive storage of lysosomal glycogen in many cell types, leading to cell and, subsequently, tissue dysfunction. Cardi...

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Bibliographic Details
Main Authors: Laurence Dubreil, Lydie Lagalice, Johan Deniaud, Antoine Sabourin, Claire Lovo, Karim Bey, Juliette Hordeaux, Chantal Thorin, Christophe Sandt, Frédéric Jamme, Marie-Anne Colle
Format: Article
Language:English
Published: IM Publications Open 2019-08-01
Series:Journal of Spectral Imaging
Subjects:
Pompe disease
glycogenosis type II
gene therapy
cardiac muscle
synchrotron microspectroscopy
infrared
glycogen
deep ­ultraviolet
tryptophan
Online Access:https://www.impopen.com/download.php?code=I08_a13

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https://www.impopen.com/download.php?code=I08_a13

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