Efficacy, pharmacokinetics, and safety in the mouse and primate retina of dual AAV vectors for Usher syndrome type 1B-Reference-Cited by-同舟云学术

Efficacy, pharmacokinetics, and safety in the mouse and primate retina of dual AAV vectors for Usher syndrome type 1B

Published:2023-03 Issue: Volume:28 Page:396-411
ISSN:2329-0501
Container-title:Molecular Therapy - Methods & Clinical Development
language:en
Short-container-title:Molecular Therapy - Methods & Clinical Development

Author:

Ferla Rita,Dell’Aquila Fabio,Doria Monica,Ferraiuolo Maria,Noto Alessia,Grazioli Fabiana,Ammendola Virginia,Testa Francesco,Melillo Paolo,Iodice Carolina,Risca Giulia,Tedesco Novella,le Brun Pierre Romain,Surace Enrico Maria,Simonelli Francesca,Galimberti Stefania,Valsecchi Maria Grazia,Marteau Jean-Brice,Veron Philippe,Colloca Stefano,Auricchio Alberto^ORCID

Publisher

Elsevier BV

Subject

Genetics,Molecular Biology,Molecular Medicine

Reference39 articles.

1. FDA approves hereditary blindness gene therapy;Nat. Biotechnol.,2018

2. Safety and vision outcomes of subretinal gene therapy targeting cone photoreceptors in achromatopsia: a nonrandomized controlled trial;Fischer;JAMA Ophthalmol.,2020

3. Choroideremia gene therapy phase 2 clinical trial: 24-month results;Lam;Am. J. Ophthalmol.,2019

4. Bilateral visual improvement with unilateral gene therapy injection for Leber hereditary optic neuropathy;Yu-Wai-Man;Sci. Transl. Med.,2020

5. Three-year follow-up of phase 1 and 2a rAAV.sFLT-1 subretinal gene therapy trials for exudative age-related macular degeneration;Rakoczy;Am. J. Ophthalmol.,2019

Cited by 7 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. Multicentric Longitudinal Prospective Study in a European Cohort of MYO7A Patients: Disease Course and Implications for Gene Therapy;Investigative Ophthalmology & Visual Science;2024-06-17

2. Elasticity and Thermal Stability are Key Determinants of Hearing Rescue by Mini-Protocadherin-15 Proteins;2024-06-17

3. Optimization strategies and advances in the research and development of AAV‐based gene therapy to deliver large transgenes;Clinical and Translational Medicine;2024-03

4. A C1qTNF3 collagen domain fusion chaperones diverse secreted proteins and anti-Aβ scFvs: Applications for gene therapies;Molecular Therapy - Methods & Clinical Development;2023-12

5. The RLR intrinsic antiviral system is expressed in neural retina and restricts lentiviral transduction of human Mueller cells;Experimental Eye Research;2023-11