Third-generation lentiviral gene therapy rescues function in a mouse model of Usher 1B-Reference-Cited by-同舟云学术

Third-generation lentiviral gene therapy rescues function in a mouse model of Usher 1B

Published:2023-12 Issue:12 Volume:31 Page:3502-3519
ISSN:1525-0016
Container-title:Molecular Therapy
language:en
Short-container-title:Molecular Therapy

Author:

Schott Juliane W.,Huang Peixin,Morgan Michael,Nelson-Brantley Jennifer,Koehler Ally,Renslo Bryan,Büning Hildegard,Warnecke Athanasia,Schambach Axel^ORCID,Staecker Hinrich

Publisher

Elsevier BV

Subject

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

Reference42 articles.

1. Reduced climbing and increased slipping adaptation in cochlear hair cells of mice with Myo7a mutations;Kros;Nat. Neurosci.,2002

2. Myosin-VIIa is expressed in multiple isoforms and essential for tensioning the hair cell mechanotransduction complex;Li;Nat. Commun.,2020

3. The autosomal recessive isolated deafness, DFNB2, and the Usher 1B syndrome are allelic defects of the myosin-VIIA gene;Weil;Nat. Genet.,1997

4. Disease mechanisms and gene therapy for Usher syndrome;Géléoc;Hear. Res.,2020

5. Clinical Presentation and Disease Course of Usher Syndrome Because of Mutations in Myo7a or Ush2a;Testa;Retina,2017

Cited by 3 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. Enhancing antitumor response by efficiently generating large-scale TCR-T cells targeting a single epitope across multiple cancer antigens;Cellular Immunology;2024-05

2. Protection from cisplatin-induced hearing loss with lentiviral vector-mediated ectopic expression of the anti-apoptotic protein BCL-XL;Molecular Therapy - Nucleic Acids;2024-03

3. CRISPR-Cas9 Engineered Extracellular Vesicles for the Treatment of Dominant Progressive Hearing Loss;2023-09-15