Treatment of infantile-onset Pompe disease in a rat model with muscle-directed AAV gene therapy-Reference-Cited by-同舟云学术

Treatment of infantile-onset Pompe disease in a rat model with muscle-directed AAV gene therapy

Published:2024-03 Issue: Volume:81 Page:101899
ISSN:2212-8778
Container-title:Molecular Metabolism
language:en
Short-container-title:Molecular Metabolism

Author:

Muñoz Sergio,Bertolin Joan,Jimenez Veronica,Jaén Maria Luisa,Garcia Miquel,Pujol Anna,Vilà Laia,Sacristan Victor,Barbon Elena,Ronzitti Giuseppe,El Andari Jihad,Tulalamba Warut,Pham Quang Hong,Ruberte Jesus,VandenDriessche Thierry,Chuah Marinee K.,Grimm Dirk,Mingozzi Federico,Bosch Fatima^ORCID

Funder

España Ministerio de Ciencia Innovación y Universidades

European Union

Generalitat de Catalunya

CIBERDEM

Publisher

Elsevier BV

Reference104 articles.

1. Pompe's disease;van der Ploeg;Lancet,2008

2. Temporal neuropathologic and behavioral phenotype of 6 neo/6 neo Pompe pisease mice;Sidman;J Neuropathol Exp Neurol,2008

3. Neural deficits contribute to respiratory insufficiency in Pompe disease;DeRuisseau;Proc Natl Acad Sci USA,2009

4. An emerging phenotype of central nervous system involvement in Pompe disease: from bench to bedside and beyond;Korlimarla;Ann Transl Med,2019

5. Pompe disease: a clinical, diagnostic, and therapeutic overview;Stevens;Curr Treat Options Neurol,2022

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1. Induced Muscle and Liver Absence of Gne in Postnatal Mice Does Not Result in Structural or Functional Muscle Impairment;Journal of Neuromuscular Diseases;2024-09-03

2. Advances in Pompe Disease Treatment: From Enzyme Replacement to Gene Therapy;Molecular Diagnosis & Therapy;2024-08-12

3. Assessing Gene Therapy Efficacy in Infantile-Onset Pompe Disease;JACC: Case Reports;2024-08

4. Adeno-associated virus vector-based gene therapy for hereditary diseases: current problems of application and approaches to solve them;Biological Products. Prevention, Diagnosis, Treatment;2024-06-20