Inclusion of a degron reduces levels of undesired inteins after AAV-mediated protein trans-splicing in the retina-Reference-Cited by-同舟云学术

Inclusion of a degron reduces levels of undesired inteins after AAV-mediated protein trans-splicing in the retina

Published:2021-12 Issue: Volume:23 Page:448-459
ISSN:2329-0501
Container-title:Molecular Therapy - Methods & Clinical Development
language:en
Short-container-title:Molecular Therapy - Methods & Clinical Development

Author:

Tornabene Patrizia^ORCID,Trapani Ivana^ORCID,Centrulo Miriam,Marrocco Elena,Minopoli Renato^ORCID,Lupo Mariangela,Iodice Carolina,Gesualdo Carlo^ORCID,Simonelli Francesca,Surace Enrico M.^ORCID,Auricchio Alberto^ORCID

Funder

European Research Council

Fondazione Telethon

Università degli Studi di Napoli Federico II

Publisher

Elsevier BV

Subject

Genetics,Molecular Biology,Molecular Medicine

Reference39 articles.

1. The new landscape of retinal gene therapy;Ku;Am. J. Med. Genet. C Semin. Med. Genet,2020

2. Efficacy, safety, and durability of voretigene neparvovec-rzyl in RPE65 mutation-associated inherited retinal dystrophy: results of phase 1 and 3 trials;Maguire;Ophthalmology,2019

3. Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial;MacLaren;Lancet,2014

4. Efficacy and safety of retinal gene therapy using adeno-associated virus vector for patients with choroideremia: a randomized clinical trial;Fischer;JAMA Ophthalmol.,2019

5. FDA approves hereditary blindness gene therapy (2018). Nat. Biotechnol. 36, 6.

Cited by 16 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. Split intein-mediated protein trans-splicing to express large dystrophins;Nature;2024-07-17

2. Safe and effective liver-directed AAV-mediated homology-independent targeted integration in mouse models of inherited diseases;Cell Reports Medicine;2024-07

3. CoCas9 is a compact nuclease from the human microbiome for efficient and precise genome editing;Nature Communications;2024-04-24

4. Adeno-associated virus as a delivery vector for gene therapy of human diseases;Signal Transduction and Targeted Therapy;2024-04-03

5. Retinal Ciliopathies and Potential Gene Therapies: A Focus on Human iPSC-Derived Organoid Models;International Journal of Molecular Sciences;2024-03-01