Literature DB >> 32561864

Retinal gene therapy: an eye-opener of the 21st century.

Anne Louise Askou1, Thomas Stax Jakobsen2, Thomas J Corydon3,4.   

Abstract

Year:  2020        PMID： 32561864     DOI： 10.1038/s41434-020-0168-2

Source DB:  PubMed          Journal:  Gene Ther        ISSN： 0969-7128            Impact factor:   5.250


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  55 in total

1.  Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65-mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial.

Authors:  Stephen Russell; Jean Bennett; Jennifer A Wellman; Daniel C Chung; Zi-Fan Yu; Amy Tillman; Janet Wittes; Julie Pappas; Okan Elci; Sarah McCague; Dominique Cross; Kathleen A Marshall; Jean Walshire; Taylor L Kehoe; Hannah Reichert; Maria Davis; Leslie Raffini; Lindsey A George; F Parker Hudson; Laura Dingfield; Xiaosong Zhu; Julia A Haller; Elliott H Sohn; Vinit B Mahajan; Wanda Pfeifer; Michelle Weckmann; Chris Johnson; Dina Gewaily; Arlene Drack; Edwin Stone; Katie Wachtel; Francesca Simonelli; Bart P Leroy; J Fraser Wright; Katherine A High; Albert M Maguire
Journal:  Lancet       Date:  2017-07-14       Impact factor: 79.321

Review 2.  Gene Therapy.

Authors:  Katherine A High; Maria G Roncarolo
Journal:  N Engl J Med       Date:  2019-08-01       Impact factor: 91.245

Review 3.  Seeing the Light after 25 Years of Retinal Gene Therapy.

Authors:  Ivana Trapani; Alberto Auricchio
Journal:  Trends Mol Med       Date:  2018-07-05       Impact factor: 11.951

4.  Two-Year Results After AAV2-Mediated Gene Therapy for Choroideremia: The Alberta Experience.

Authors:  Ioannis S Dimopoulos; Stephanie C Hoang; Alina Radziwon; Natalia M Binczyk; Miguel C Seabra; Robert E MacLaren; Rizwan Somani; Matthew T S Tennant; Ian M MacDonald
Journal:  Am J Ophthalmol       Date:  2018-06-27       Impact factor: 5.258

5.  Safety of rAAV2/2-ND4 Gene Therapy for Leber Hereditary Optic Neuropathy.

Authors:  Catherine Vignal; Scott Uretsky; Serge Fitoussi; Anne Galy; Laure Blouin; Jean-François Girmens; Samuel Bidot; Nitza Thomasson; Céline Bouquet; Sonia Valero; Sandrine Meunier; Jean-Philippe Combal; Bernard Gilly; Barrett Katz; José-Alain Sahel
Journal:  Ophthalmology       Date:  2018-02-14       Impact factor: 12.079

Review 6.  Gene therapy for inherited retinal and optic nerve degenerations.

Authors:  Nicholas A Moore; Nuria Morral; Thomas A Ciulla; Peter Bracha
Journal:  Expert Opin Biol Ther       Date:  2017-10-23       Impact factor: 4.388

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.  Has retinal gene therapy come of age? From bench to bedside and back to bench.

Authors:  Ivana Trapani; Alberto Auricchio
Journal:  Hum Mol Genet       Date:  2019-10-01       Impact factor: 6.150

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

10.  Choroideremia Gene Therapy Phase 2 Clinical Trial: 24-Month Results.

Authors:  Byron L Lam; Janet L Davis; Ninel Z Gregori; Robert E MacLaren; Aniz Girach; Jennifer D Verriotto; Belen Rodriguez; Potyra R Rosa; Xiaojun Zhang; William J Feuer
Journal:  Am J Ophthalmol       Date:  2018-09-19       Impact factor: 5.258
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  8 in total

Review 1.  Retinal Glutamate Neurotransmission: From Physiology to Pathophysiological Mechanisms of Retinal Ganglion Cell Degeneration.

Authors:  Isabella Boccuni; Richard Fairless
Journal:  Life (Basel)       Date:  2022-04-25

Review 2.  Associations between the Complement System and Choroidal Neovascularization in Wet Age-Related Macular Degeneration.

Authors:  Emilie Grarup Jensen; Thomas Stax Jakobsen; Steffen Thiel; Anne Louise Askou; Thomas J Corydon
Journal:  Int J Mol Sci       Date:  2020-12-21       Impact factor: 5.923

Review 3.  Experimental Therapeutic Approaches for the Treatment of Retinal Pathology in Neuronal Ceroid Lipofuscinoses.

Authors:  Udo Bartsch; Stephan Storch
Journal:  Front Neurol       Date:  2022-04-18       Impact factor: 4.003

Review 4.  Retinal Ciliopathy in the Patient with Transplanted Kidney: Case Report.

Authors:  Ivona Bućan; Mirjana Bjeloš; Irena Marković; Diana Bućan
Journal:  Int J Mol Sci       Date:  2022-07-08       Impact factor: 6.208

5.  Trials and tribulations of rare eye diseases.

Authors:  Rohit Shetty; Swaminathan Sethu; Chaitra Jayadev
Journal:  Indian J Ophthalmol       Date:  2022-07       Impact factor: 2.969

6.  Photodisruption of the Inner Limiting Membrane: Exploring ICG Loaded Nanoparticles as Photosensitizers.

Authors:  Kaat De Clerck; Geraldine Accou; Félix Sauvage; Kevin Braeckmans; Stefaan C De Smedt; Katrien Remaut; Karen Peynshaert
Journal:  Pharmaceutics       Date:  2022-08-17       Impact factor: 6.525

7.  Neuroprotective Gene Therapy by Overexpression of the Transcription Factor MAX in Rat Models of Glaucomatous Neurodegeneration.

Authors:  Rafael Lani-Louzada; Camila Marra; Mariana Santana Dias; Victor Guedes de Araújo; Carla Andreia Abreu; Vinícius Toledo Ribas; Daniel Adesse; Silvana Allodi; Vince Chiodo; William Hauswirth; Hilda Petrs-Silva; Rafael Linden
Journal:  Invest Ophthalmol Vis Sci       Date:  2022-02-01       Impact factor: 4.799

8.  Targeted Knockout of the Vegfa Gene in the Retina by Subretinal Injection of RNP Complexes Containing Cas9 Protein and Modified sgRNAs.

Authors:  Andreas Braae Holmgaard; Anne Louise Askou; Emilie Grarup Jensen; Sidsel Alsing; Rasmus O Bak; Jacob Giehm Mikkelsen; Thomas J Corydon
Journal:  Mol Ther       Date:  2020-09-23       Impact factor: 11.454
  8 in total

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