Literature DB >> 31884598

Progress in Gene Therapy for Rhodopsin Autosomal Dominant Retinitis Pigmentosa.

Raghavi Sudharsan1, William A Beltran2.   

Abstract

This brief review summarizes the major proof-of-concept gene therapy studies for autosomal dominant retinitis pigmentosa (RP) caused by mutations in the rhodopsin gene (RHO-adRP) that have been conducted over the past 20 years in various animal models. We have listed in tabular form the various approaches, gene silencing reagents, gene delivery strategies, and salient results from these studies.

Entities:  

Keywords:  Autosomal dominant retinitis pigmentosa; Gene therapy; Knockdown and replacement; Rhodpsin

Mesh：

Substances：

Year:  2019        PMID： 31884598      PMCID： PMC7217593          DOI： 10.1007/978-3-030-27378-1_19

Source DB:  PubMed          Journal:  Adv Exp Med Biol        ISSN： 0065-2598            Impact factor:   2.622


  22 in total

1.  Toward a gene therapy for dominant disease: validation of an RNA interference-based mutation-independent approach.

Authors:  Anna-Sophia Kiang; Arpad Palfi; Marius Ader; Paul F Kenna; Sophia Millington-Ward; Gerry Clark; Avril Kennan; Mary O'reilly; Lawrence C T Tam; Aileen Aherne; Niamh McNally; Pete Humphries; G Jane Farrar
Journal:  Mol Ther       Date:  2005-09       Impact factor: 11.454

2.  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

3.  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

4.  Suppression of mouse rhodopsin expression in vivo by AAV mediated siRNA delivery.

Authors:  M Gorbatyuk; V Justilien; J Liu; W W Hauswirth; A S Lewin
Journal:  Vision Res       Date:  2007-02-12       Impact factor: 1.886

5.  Ribozyme rescue of photoreceptor cells in a transgenic rat model of autosomal dominant retinitis pigmentosa.

Authors:  A S Lewin; K A Drenser; W W Hauswirth; S Nishikawa; D Yasumura; J G Flannery; M M LaVail
Journal:  Nat Med       Date:  1998-08       Impact factor: 53.440

6.  A transgenic mouse model for gene therapy of rhodopsin-linked Retinitis Pigmentosa.

Authors:  Mary O'Reilly; Sophia Millington-Ward; Arpad Palfi; Naomi Chadderton; Thérèse Cronin; Niamh McNally; Marian M Humphries; Peter Humphries; Paul F Kenna; G Jane Farrar
Journal:  Vision Res       Date:  2007-10-24       Impact factor: 1.886

7.  Preferential silencing of a common dominant rhodopsin mutation does not inhibit retinal degeneration in a transgenic model.

Authors:  Alessandra Tessitore; Fabiana Parisi; Michela Alessandra Denti; Mariacarmela Allocca; Umberto Di Vicino; Luciano Domenici; Irene Bozzoni; Alberto Auricchio
Journal:  Mol Ther       Date:  2006-09-18       Impact factor: 11.454

8.  RNA interference-mediated suppression and replacement of human rhodopsin in vivo.

Authors:  Mary O'Reilly; Arpad Palfi; Naomi Chadderton; Sophia Millington-Ward; Marius Ader; Thérèse Cronin; Thérèse Tuohy; Alberto Auricchio; Markus Hildinger; Amanda Tivnan; Niamh McNally; Marian M Humphries; Anna-Sophia Kiang; Pete Humphries; Paul F Kenna; G Jane Farrar
Journal:  Am J Hum Genet       Date:  2007-05-23       Impact factor: 11.025

9.  In Vivo CRISPR/Cas9 Gene Editing Corrects Retinal Dystrophy in the S334ter-3 Rat Model of Autosomal Dominant Retinitis Pigmentosa.

Authors:  Benjamin Bakondi; Wenjian Lv; Bin Lu; Melissa K Jones; Yuchun Tsai; Kevin J Kim; Rachelle Levy; Aslam Abbasi Akhtar; Joshua J Breunig; Clive N Svendsen; Shaomei Wang
Journal:  Mol Ther       Date:  2015-12-15       Impact factor: 11.454

10.  In vivo Editing of the Human Mutant Rhodopsin Gene by Electroporation of Plasmid-based CRISPR/Cas9 in the Mouse Retina.

Authors:  Maria Carmela Latella; Maria Teresa Di Salvo; Fabienne Cocchiarella; Daniela Benati; Giulia Grisendi; Antonella Comitato; Valeria Marigo; Alessandra Recchia
Journal:  Mol Ther Nucleic Acids       Date:  2016-11-22       Impact factor: 10.183
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  4 in total

1.  CRISPR genome surgery in a novel humanized model for autosomal dominant retinitis pigmentosa.

Authors:  Wen-Hsuan Wu; Yi-Ting Tsai; I-Wen Huang; Chia-Hua Cheng; Chun-Wei Hsu; Xuan Cui; Joseph Ryu; Peter M J Quinn; Salvatore Marco Caruso; Chyuang-Sheng Lin; Stephen H Tsang
Journal:  Mol Ther       Date:  2022-02-10       Impact factor: 12.910

Review 2.  Pluripotent stem cell therapy for retinal diseases.

Authors:  Ishrat Ahmed; Robert J Johnston; Mandeep S Singh
Journal:  Ann Transl Med       Date:  2021-08

3.  CRISPR DNA Base Editing Strategies for Treating Retinitis Pigmentosa Caused by Mutations in Rhodopsin.

Authors:  Maria Kaukonen; Michelle E McClements; Robert E MacLaren
Journal:  Genes (Basel)       Date:  2022-07-26       Impact factor: 4.141

4.  Sector Retinitis Pigmentosa: Extending the Molecular Genetics Basis and Elucidating the Natural History.

Authors:  Michalis Georgiou; Parampal S Grewal; Akshay Narayan; Muath Alser; Naser Ali; Kaoru Fujinami; Andrew R Webster; Michel Michaelides
Journal:  Am J Ophthalmol       Date:  2020-08-12       Impact factor: 5.258
  4 in total

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