Literature DB >> 28391961

Taking Stock of Retinal Gene Therapy: Looking Back and Moving Forward.

Jean Bennett1.   

Abstract

Over the past 20 years, there has been tremendous progress in retinal gene therapy. The safety and efficacy results in one early-onset severe blinding disease may lead to the first gene therapy drug approval in the United States. Here, we review how far the field has come over the past two decades and speculate on the directions that the field will take in the future.
Copyright © 2017 The American Society of Gene and Cell Therapy. Published by Elsevier Inc. All rights reserved.

Entities:  

Keywords:  clinical trials; gene therapy; retina

Mesh：

Substances：

Year:  2017        PMID： 28391961      PMCID： PMC5417792          DOI： 10.1016/j.ymthe.2017.03.008

Source DB:  PubMed          Journal:  Mol Ther        ISSN： 1525-0016            Impact factor:   12.910


  129 in total

1.  Autosomal dominant retinitis pigmentosa: a mutation in codon 178 of the rhodopsin gene in two families of Celtic origin.

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Journal:  Genomics       Date:  1991-12       Impact factor: 5.736

2.  High-efficiency transduction of the mouse retina by tyrosine-mutant AAV serotype vectors.

Authors:  Hilda Petrs-Silva; Astra Dinculescu; Qiuhong Li; Seok-Hong Min; Vince Chiodo; Ji-Jing Pang; Li Zhong; Sergei Zolotukhin; Arun Srivastava; Alfred S Lewin; William W Hauswirth
Journal:  Mol Ther       Date:  2008-12-16       Impact factor: 11.454

3.  Rom-1 is required for rod photoreceptor viability and the regulation of disk morphogenesis.

Authors:  G Clarke; A F Goldberg; D Vidgen; L Collins; L Ploder; L Schwarz; L L Molday; J Rossant; A Szél; R S Molday; D G Birch; R R McInnes
Journal:  Nat Genet       Date:  2000-05       Impact factor: 38.330

4.  Adenoviral vector-delivered pigment epithelium-derived factor for neovascular age-related macular degeneration: results of a phase I clinical trial.

Authors:  Peter A Campochiaro; Quan Dong Nguyen; Syed Mahmood Shah; Michael L Klein; Eric Holz; Robert N Frank; David A Saperstein; Anurag Gupta; J Timothy Stout; Jennifer Macko; Robert DiBartolomeo; Lisa L Wei
Journal:  Hum Gene Ther       Date:  2006-02       Impact factor: 5.695

5.  A point mutation of the rhodopsin gene in one form of retinitis pigmentosa.

Authors:  T P Dryja; T L McGee; E Reichel; L B Hahn; G S Cowley; D W Yandell; M A Sandberg; E L Berson
Journal:  Nature       Date:  1990-01-25       Impact factor: 49.962

6.  Genomic DNA nanoparticles rescue rhodopsin-associated retinitis pigmentosa phenotype.

Authors:  Zongchao Han; Marcellus J Banworth; Rasha Makkia; Shannon M Conley; Muayyad R Al-Ubaidi; Mark J Cooper; Muna I Naash
Journal:  FASEB J       Date:  2015-02-24       Impact factor: 5.191

7.  Treatment of retinitis pigmentosa due to MERTK mutations by ocular subretinal injection of adeno-associated virus gene vector: results of a phase I trial.

Authors:  Nicola G Ghazi; Emad B Abboud; Sawsan R Nowilaty; Hisham Alkuraya; Abdulrahman Alhommadi; Huimin Cai; Rui Hou; Wen-Tao Deng; Sanford L Boye; Abdulrahman Almaghamsi; Fahad Al Saikhan; Hassan Al-Dhibi; David Birch; Christopher Chung; Dilek Colak; Matthew M LaVail; Douglas Vollrath; Kirsten Erger; Wenqiu Wang; Thomas Conlon; Kang Zhang; William Hauswirth; Fowzan S Alkuraya
Journal:  Hum Genet       Date:  2016-01-29       Impact factor: 4.132

Review 8.  Worldwide Argus II implantation: recommendations to optimize patient outcomes.

Authors:  Devon H Ghodasra; Adrienne Chen; J Fernando Arevalo; David G Birch; Kari Branham; Brian Coley; Gislin Dagnelie; Eugene de Juan; Robert G Devenyi; Jessy D Dorn; Andy Fisher; Duane R Geruschat; Ninel Z Gregori; Robert J Greenberg; Paul Hahn; Allen C Ho; Ashley Howson; Suber S Huang; Raymond Iezzi; Naheed Khan; Byron L Lam; Jennifer I Lim; Kirsten G Locke; Michelle Markowitz; Anne-Marie Ripley; Mark Rankin; Hannah Schimitzek; Fay Tripp; James D Weiland; Jiong Yan; David N Zacks; K Thiran Jayasundera
Journal:  BMC Ophthalmol       Date:  2016-05-06       Impact factor: 2.209

9.  Novel adeno-associated virus serotypes efficiently transduce murine photoreceptors.

Authors:  Mariacarmela Allocca; Claudio Mussolino; Maria Garcia-Hoyos; Daniela Sanges; Carolina Iodice; Marco Petrillo; Luk H Vandenberghe; James M Wilson; Valeria Marigo; Enrico M Surace; Alberto Auricchio
Journal:  J Virol       Date:  2007-08-15       Impact factor: 5.103

10.  Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2 clinical trial.

Authors:  Robert E MacLaren; Markus Groppe; Alun R Barnard; Charles L Cottriall; Tanya Tolmachova; Len Seymour; K Reed Clark; Matthew J During; Frans P M Cremers; Graeme C M Black; Andrew J Lotery; Susan M Downes; Andrew R Webster; Miguel C Seabra
Journal:  Lancet       Date:  2014-01-16       Impact factor: 79.321
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  35 in total

1.  Complement C3-Targeted Gene Therapy Restricts Onset and Progression of Neurodegeneration in Chronic Mouse Glaucoma.

Authors:  Alejandra Bosco; Sarah R Anderson; Kevin T Breen; Cesar O Romero; Michael R Steele; Vince A Chiodo; Sanford L Boye; William W Hauswirth; Stephen Tomlinson; Monica L Vetter
Journal:  Mol Ther       Date:  2018-08-24       Impact factor: 11.454

2.  Reference Standards for Gene and Cell Therapy Products.

Authors:  Boro Dropulić
Journal:  Mol Ther       Date:  2017-05-11       Impact factor: 11.454

3.  Retina transduction by rAAV2 after intravitreal injection: comparison between mouse and rat.

Authors:  Mariana S Dias; Victor G Araujo; Taliane Vasconcelos; Qiuhong Li; William W Hauswirth; Rafael Linden; Hilda Petrs-Silva
Journal:  Gene Ther       Date:  2019-09-27       Impact factor: 5.250

4.  Circular RNA-ZNF532 regulates diabetes-induced retinal pericyte degeneration and vascular dysfunction.

Authors:  Qin Jiang; Chang Liu; Chao-Peng Li; Shan-Shan Xu; Mu-Di Yao; Hui-Min Ge; Ya-Nan Sun; Xiu-Miao Li; Shu-Jie Zhang; Kun Shan; Bai-Hui Liu; Jin Yao; Chen Zhao; Biao Yan
Journal:  J Clin Invest       Date:  2020-07-01       Impact factor: 14.808

5.  The primate model for understanding and restoring vision.

Authors:  Serge Picaud; Deniz Dalkara; Katia Marazova; Olivier Goureau; Botond Roska; José-Alain Sahel
Journal:  Proc Natl Acad Sci U S A       Date:  2019-12-23       Impact factor: 11.205

6.  Retinal Tropism and Transduction of Adeno-Associated Virus Varies by Serotype and Route of Delivery (Intravitreal, Subretinal, or Suprachoroidal) in Rats.

Authors:  Ian C Han; Justine L Cheng; Erin R Burnight; Christy L Ralston; Jessica L Fick; Gabriella J Thomsen; Emilio F Tovar; Stephen R Russell; Elliott H Sohn; Robert F Mullins; Edwin M Stone; Budd A Tucker; Luke A Wiley
Journal:  Hum Gene Ther       Date:  2020-10-20       Impact factor: 5.695

Review 7.  Restoring Vision to the Blind with Chemical Photoswitches.

Authors:  Ivan Tochitsky; Michael A Kienzler; Ehud Isacoff; Richard H Kramer
Journal:  Chem Rev       Date:  2018-06-06       Impact factor: 60.622

8.  Amelioration of Neurosensory Structure and Function in Animal and Cellular Models of a Congenital Blindness.

Authors:  Ji Yun Song; Puya Aravand; Sergei Nikonov; Lanfranco Leo; Arkady Lyubarsky; Jeannette L Bennicelli; Jieyan Pan; Zhangyong Wei; Ivan Shpylchak; Pamela Herrera; Daniel J Bennett; Nicoletta Commins; Albert M Maguire; Jennifer Pham; Anneke I den Hollander; Frans P M Cremers; Robert K Koenekoop; Ronald Roepman; Patsy Nishina; Shangzhen Zhou; Wei Pan; Gui-Shuang Ying; Tomas S Aleman; Jimmy de Melo; Ilan McNamara; Junwei Sun; Jason Mills; Jean Bennett
Journal:  Mol Ther       Date:  2018-03-21       Impact factor: 11.454

9.  Mutation-independent rhodopsin gene therapy by knockdown and replacement with a single AAV vector.

Authors:  Artur V Cideciyan; Raghavi Sudharsan; Valérie L Dufour; Michael T Massengill; Simone Iwabe; Malgorzata Swider; Brianna Lisi; Alexander Sumaroka; Luis Felipe Marinho; Tatyana Appelbaum; Brian Rossmiller; William W Hauswirth; Samuel G Jacobson; Alfred S Lewin; Gustavo D Aguirre; William A Beltran
Journal:  Proc Natl Acad Sci U S A       Date:  2018-08-20       Impact factor: 11.205

Review 10.  A Systematic Review and Meta-Analyses of Interventional Clinical Trial Studies for Gene Therapies for the Inherited Retinal Degenerations (IRDs).

Authors:  Gearóid P Tuohy; Roly Megaw
Journal:  Biomolecules       Date:  2021-05-19
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