Precision medicine in action for Pompe disease-Reference-Cited by-同舟云学术

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Precision medicine in action for Pompe disease

Published:2024-09 Issue:3 Volume:35 Page:102265
ISSN:2162-2531
Container-title:Molecular Therapy - Nucleic Acids
language:en
Short-container-title:Molecular Therapy - Nucleic Acids

Author:

Tarallo Antonietta,Parenti Giancarlo,Brunetti-Pierri Nicola^ORCID

Publisher

Elsevier BV

Reference8 articles.

1. The emerging phenotype of long-term survivors with infantile Pompe disease;Prater;Genet. Med.,2012

2. Gene therapy for glycogen storage diseases;Koeberl;J. Inherit. Metab. Dis.,2024

3. Base editing rescues acid α-glucosidase function in infantile-onset Pompe disease patient-derived cells;Christensen;Mol. Ther. Nucleic Acids,2024

4. Adenine base editing in mouse embryos and an adult mouse model of Duchenne muscular dystrophy;Ryu;Nat. Biotechnol.,2018

5. Cytosine and adenine base editing of the brain, liver, retina, heart and skeletal muscle of mice via adeno-associated viruses;Levy;Nat. Biomed. Eng.,2020

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