Functional Recovery in a Friedreich's Ataxia Mouse Model by Frataxin Gene Transfer Using an HSV-1 Amplicon Vector-Reference-Cited by-同舟云学术

Functional Recovery in a Friedreich's Ataxia Mouse Model by Frataxin Gene Transfer Using an HSV-1 Amplicon Vector

Published:2007-06 Issue:6 Volume:15 Page:1072-1078
ISSN:1525-0016
Container-title:Molecular Therapy
language:en
Short-container-title:Molecular Therapy

Author:

Lim Filip,Palomo Gloria M,Mauritz Christina,Giménez-Cassina Alfredo,Illana Belen,Wandosell Francisco,Díaz-Nido Javier

Publisher

Elsevier BV

Subject

Drug Discovery,Pharmacology,Genetics,Molecular Biology,Molecular Medicine

Reference38 articles.

1. Friedreich's ataxia: autosomal recessive disease caused by an intronic GAA triplet repeat expansion;Campuzano;Science,1996

2. Identification of a novel transcript of X25, the human gene involved in Friedreich's ataxia;Pianese;Neurosci Lett,2002

3. Identification of a missense mutation in a Friedreich's ataxia patient: implications for diagnosis and carrier studies;Bartolo;Am J Med Genet,1998

4. Atypical Friedreich ataxia caused by compound heterozygosity for a novel missense mutation and the GAA triplet-repeat expansion;Bidichandani;Am J Hum Genet,1997

5. Friedreich's ataxia: point mutations and clinical presentation of compound heterozygotes;Cossee;Ann Neurol,1999

Cited by 49 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. Patient-derived iPSC models of Friedreich ataxia: a new frontier for understanding disease mechanisms and therapeutic application;Translational Neurodegeneration;2023-09-20

2. Recent Advances on Therapeutic Approaches for Friedreich’s Ataxia: New Pharmacological Targets, Protein, and Gene Therapy;Contemporary Clinical Neuroscience;2023

3. Frataxin deficiency alters gene expression in Friedreich ataxia derived IPSC‐neurons and cardiomyocytes;Molecular Genetics & Genomic Medicine;2022-11-11

4. Advantages and Limitations of Gene Therapy and Gene Editing for Friedreich’s Ataxia;Frontiers in Genome Editing;2022-05-17

5. Cerebellar Pathology in an Inducible Mouse Model of Friedreich Ataxia;Frontiers in Neuroscience;2022-03-24