Literature DB >> 30569401

Attenuation of Inherited and Acquired Retinal Degeneration Progression with Gene-based Techniques.

Galaxy Y Cho1,2,3, Kyle Bolo4, Karen Sophia Park1,2, Jesse D Sengillo5, Stephen H Tsang6,7,8,9,10.   

Abstract

Inherited retinal dystrophies cause progressive vision loss and are major contributors to blindness worldwide. Advances in gene therapy have brought molecular approaches into the realm of clinical trials for these incurable illnesses. Select phase I, II and III trials are complete and provide some promise in terms of functional outcomes and safety, although questions do remain over the durability of their effects and the prevalence of inflammatory reactions. This article reviews gene therapy as it can be applied to inherited retinal dystrophies, provides an update of results from recent clinical trials, and discusses the future prospects of gene therapy and genome surgery.

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Mesh：

Year:  2019        PMID： 30569401      PMCID： PMC6380912          DOI： 10.1007/s40291-018-0377-1

Source DB:  PubMed          Journal:  Mol Diagn Ther        ISSN： 1177-1062            Impact factor:   4.074


  89 in total

Review 1.  Adeno-associated Virus as a Mammalian DNA Vector.

Authors:  Max Salganik; Matthew L Hirsch; Richard Jude Samulski
Journal:  Microbiol Spectr       Date:  2015-08

2.  Highly Efficient Delivery of Adeno-Associated Viral Vectors to the Primate Retina.

Authors:  Shannon E Boye; John J Alexander; C Douglas Witherspoon; Sanford L Boye; James J Peterson; Mark E Clark; Kristen J Sandefer; Chris A Girkin; William W Hauswirth; Paul D Gamlin
Journal:  Hum Gene Ther       Date:  2016-08       Impact factor: 5.695

3.  Gene Therapy in Neovascular Age-related Macular Degeneration: Three-Year Follow-up of a Phase 1 Randomized Dose Escalation Trial.

Authors:  Ian J Constable; Chooi-May Lai; Aaron L Magno; Martyn A French; Samuel B Barone; Steven D Schwartz; Mark S Blumenkranz; Mariapia A Degli-Esposti; Elizabeth P Rakoczy
Journal:  Am J Ophthalmol       Date:  2017-02-27       Impact factor: 5.258

Review 4.  Adeno-associated virus at 50: a golden anniversary of discovery, research, and gene therapy success--a personal perspective.

Authors:  Eric Hastie; R Jude Samulski
Journal:  Hum Gene Ther       Date:  2015-04-20       Impact factor: 5.695

5.  Human retinal gene therapy for Leber congenital amaurosis shows advancing retinal degeneration despite enduring visual improvement.

Authors:  Artur V Cideciyan; Samuel G Jacobson; William A Beltran; Alexander Sumaroka; Malgorzata Swider; Simone Iwabe; Alejandro J Roman; Melani B Olivares; Sharon B Schwartz; András M Komáromy; William W Hauswirth; Gustavo D Aguirre
Journal:  Proc Natl Acad Sci U S A       Date:  2013-01-22       Impact factor: 11.205

6.  Safety and efficacy of gene transfer for Leber's congenital amaurosis.

Authors:  Albert M Maguire; Francesca Simonelli; Eric A Pierce; Edward N Pugh; Federico Mingozzi; Jeannette Bennicelli; Sandro Banfi; Kathleen A Marshall; Francesco Testa; Enrico M Surace; Settimio Rossi; Arkady Lyubarsky; Valder R Arruda; Barbara Konkle; Edwin Stone; Junwei Sun; Jonathan Jacobs; Lou Dell'Osso; Richard Hertle; Jian-xing Ma; T Michael Redmond; Xiaosong Zhu; Bernd Hauck; Olga Zelenaia; Kenneth S Shindler; Maureen G Maguire; J Fraser Wright; Nicholas J Volpe; Jennifer Wellman McDonnell; Alberto Auricchio; Katherine A High; Jean Bennett
Journal:  N Engl J Med       Date:  2008-04-27       Impact factor: 91.245

7.  Safety and durability of effect of contralateral-eye administration of AAV2 gene therapy in patients with childhood-onset blindness caused by RPE65 mutations: a follow-on phase 1 trial.

Authors:  Jean Bennett; Jennifer Wellman; Kathleen A Marshall; Sarah McCague; Manzar Ashtari; Julie DiStefano-Pappas; Okan U Elci; Daniel C Chung; Junwei Sun; J Fraser Wright; Dominique R Cross; Puya Aravand; Laura L Cyckowski; Jeannette L Bennicelli; Federico Mingozzi; Alberto Auricchio; Eric A Pierce; Jason Ruggiero; Bart P Leroy; Francesca Simonelli; Katherine A High; Albert M Maguire
Journal:  Lancet       Date:  2016-06-30       Impact factor: 79.321

Review 8.  The Future Looks Brighter After 25 Years of Retinal Gene Therapy.

Authors:  Alberto Auricchio; Alexander J Smith; Robin R Ali
Journal:  Hum Gene Ther       Date:  2017-08-20       Impact factor: 5.695

Review 9.  Leber hereditary optic neuropathy: current perspectives.

Authors:  Cherise Meyerson; Greg Van Stavern; Collin McClelland
Journal:  Clin Ophthalmol       Date:  2015-06-26

10.  Retrospective Analysis of Structural Disease Progression in Retinitis Pigmentosa Utilizing Multimodal Imaging.

Authors:  Thiago Cabral; Jesse D Sengillo; Jimmy K Duong; Sally Justus; Katherine Boudreault; Kaspar Schuerch; Rubens Belfort; Vinit B Mahajan; Janet R Sparrow; Stephen H Tsang
Journal:  Sci Rep       Date:  2017-09-04       Impact factor: 4.379
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  3 in total

1.  Comparative Analysis of Functional and Structural Decline in Retinitis Pigmentosas.

Authors:  Thiago Cabral; Jose Ronaldo Lima de Carvalho; Joonpyo Kim; Jin Kyun Oh; Sarah R Levi; Karen Sophia Park; Jimmy K Duong; Junhyung Park; Katherine Boudreault; Rubens Belfort; Stephen H Tsang
Journal:  Int J Mol Sci       Date:  2020-04-15       Impact factor: 5.923

2.  Learning to see again: Perceptual learning of simulated abnormal on- off-cell population responses in sighted individuals.

Authors:  Rebecca B Esquenazi; Kimberly Meier; Michael Beyeler; Geoffrey M Boynton; Ione Fine
Journal:  J Vis       Date:  2021-12-01       Impact factor: 2.240

Review 3.  Gene-Based Therapeutics for Acquired Retinal Disease: Opportunities and Progress.

Authors:  Tien-En Tan; Beau James Fenner; Veluchamy Amutha Barathi; Sai Bo Bo Tun; Yeo Sia Wey; Andrew Shih Hsiang Tsai; Xinyi Su; Shu Yen Lee; Chui Ming Gemmy Cheung; Tien Yin Wong; Jodhbir Singh Mehta; Kelvin Yi Chong Teo
Journal:  Front Genet       Date:  2021-12-07       Impact factor: 4.599
  3 in total

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