Literature DB >> 32719823

Correcting visual loss by genetics and prosthetics.

Kanmin Xue1, Robert E MacLaren1.   

Abstract

Retinal degenerations account for the majority of untreatable causes of blindness. Advances in gene delivery vectors, CRISPR/Cas9-based gene editing systems, and electronic engineering have led to a wide range of strategies for correcting visual loss. Here, we provide an overview of retinal gene therapy, gene editing, optogenetics and retinal prostheses using examples from recent clinical trials and pre-clinical studies.

Entities:  

Year:  2020        PMID: 32719823      PMCID: PMC7115840          DOI: 10.1016/j.cophys.2020.03.003

Source DB:  PubMed          Journal:  Curr Opin Physiol        ISSN: 2468-8673


  42 in total

1.  Retinal AAV8-RS1 Gene Therapy for X-Linked Retinoschisis: Initial Findings from a Phase I/IIa Trial by Intravitreal Delivery.

Authors:  Catherine Cukras; Henry E Wiley; Brett G Jeffrey; H Nida Sen; Amy Turriff; Yong Zeng; Camasamudram Vijayasarathy; Dario Marangoni; Lucia Ziccardi; Sten Kjellstrom; Tae Kwon Park; Suja Hiriyanna; J Fraser Wright; Peter Colosi; Zhijian Wu; Ronald A Bush; Lisa L Wei; Paul A Sieving
Journal:  Mol Ther       Date:  2018-07-07       Impact factor: 11.454

2.  LiGluR restores visual responses in rodent models of inherited blindness.

Authors:  Natalia Caporale; Kathleen D Kolstad; Trevor Lee; Ivan Tochitsky; Deniz Dalkara; Dirk Trauner; Richard Kramer; Yang Dan; Ehud Y Isacoff; John G Flannery
Journal:  Mol Ther       Date:  2011-05-24       Impact factor: 11.454

3.  Five-Year Safety and Performance Results from the Argus II Retinal Prosthesis System Clinical Trial.

Authors:  Lyndon da Cruz; Jessy D Dorn; Mark S Humayun; Gislin Dagnelie; James Handa; Pierre-Olivier Barale; José-Alain Sahel; Paulo E Stanga; Farhad Hafezi; Avinoam B Safran; Joel Salzmann; Arturo Santos; David Birch; Rand Spencer; Artur V Cideciyan; Eugene de Juan; Jacque L Duncan; Dean Eliott; Amani Fawzi; Lisa C Olmos de Koo; Allen C Ho; Gary Brown; Julia Haller; Carl Regillo; Lucian V Del Priore; Aries Arditi; Robert J Greenberg
Journal:  Ophthalmology       Date:  2016-07-21       Impact factor: 12.079

4.  Cloning of adeno-associated virus into pBR322: rescue of intact virus from the recombinant plasmid in human cells.

Authors:  R J Samulski; K I Berns; M Tan; N Muzyczka
Journal:  Proc Natl Acad Sci U S A       Date:  1982-03       Impact factor: 11.205

5.  Age-dependent effects of RPE65 gene therapy for Leber's congenital amaurosis: a phase 1 dose-escalation trial.

Authors:  Albert M Maguire; Katherine A High; Alberto Auricchio; J Fraser Wright; Eric A Pierce; Francesco Testa; Federico Mingozzi; Jeannette L Bennicelli; Gui-shuang Ying; Settimio Rossi; Ann Fulton; Kathleen A Marshall; Sandro Banfi; Daniel C Chung; Jessica I W Morgan; Bernd Hauck; Olga Zelenaia; Xiaosong Zhu; Leslie Raffini; Frauke Coppieters; Elfride De Baere; Kenneth S Shindler; Nicholas J Volpe; Enrico M Surace; Carmela Acerra; Arkady Lyubarsky; T Michael Redmond; Edwin Stone; Junwei Sun; Jennifer Wellman McDonnell; Bart P Leroy; Francesca Simonelli; Jean Bennett
Journal:  Lancet       Date:  2009-10-23       Impact factor: 79.321

6.  In vivo-directed evolution of a new adeno-associated virus for therapeutic outer retinal gene delivery from the vitreous.

Authors:  Deniz Dalkara; Leah C Byrne; Ryan R Klimczak; Meike Visel; Lu Yin; William H Merigan; John G Flannery; David V Schaffer
Journal:  Sci Transl Med       Date:  2013-06-12       Impact factor: 17.956

7.  Interim Results of a Multicenter Trial with the New Electronic Subretinal Implant Alpha AMS in 15 Patients Blind from Inherited Retinal Degenerations.

Authors:  Katarina Stingl; Ruth Schippert; Karl U Bartz-Schmidt; Dorothea Besch; Charles L Cottriall; Thomas L Edwards; Florian Gekeler; Udo Greppmaier; Katja Kiel; Assen Koitschev; Laura Kühlewein; Robert E MacLaren; James D Ramsden; Johann Roider; Albrecht Rothermel; Helmut Sachs; Greta S Schröder; Jan Tode; Nicole Troelenberg; Eberhart Zrenner
Journal:  Front Neurosci       Date:  2017-08-23       Impact factor: 4.677

Review 8.  A systems biology approach towards understanding and treating non-neovascular age-related macular degeneration.

Authors:  James T Handa; Cathy Bowes Rickman; Andrew D Dick; Michael B Gorin; Joan W Miller; Cynthia A Toth; Marius Ueffing; Marco Zarbin; Lindsay A Farrer
Journal:  Nat Commun       Date:  2019-07-26       Impact factor: 14.919

9.  Beneficial effects on vision in patients undergoing retinal gene therapy for choroideremia.

Authors:  Kanmin Xue; Jasleen K Jolly; Alun R Barnard; Anna Rudenko; Anna P Salvetti; Maria I Patrício; Thomas L Edwards; Markus Groppe; Harry O Orlans; Tanya Tolmachova; Graeme C Black; Andrew R Webster; Andrew J Lotery; Graham E Holder; Susan M Downes; Miguel C Seabra; Robert E MacLaren
Journal:  Nat Med       Date:  2018-10-08       Impact factor: 53.440

Review 10.  The promise and challenge of therapeutic genome editing.

Authors:  Jennifer A Doudna
Journal:  Nature       Date:  2020-02-12       Impact factor: 49.962

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  1 in total

Review 1.  Clinical Progress and Optimization of Information Processing in Artificial Visual Prostheses.

Authors:  Jing Wang; Rongfeng Zhao; Peitong Li; Zhiqiang Fang; Qianqian Li; Yanling Han; Ruyan Zhou; Yun Zhang
Journal:  Sensors (Basel)       Date:  2022-08-30       Impact factor: 3.847

  1 in total

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