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Literature DB >> 23458444

Retinal gene therapy coming of age.

Connie L Cepko¹, Luk H Vandenberghe.

Abstract

Year: 2013 PMID： 23458444 PMCID： PMC4530353 DOI： 10.1089/hum.2013.050

Source DB: PubMed Journal: Hum Gene Ther ISSN： 1043-0342 Impact factor: 5.695

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13 in total

Review 1. Retinitis pigmentosa.

Authors: Dyonne T Hartong; Eliot L Berson; Thaddeus P Dryja
Journal: Lancet Date: 2006-11-18 Impact factor: 79.321

2. Non-cell-autonomous photoreceptor degeneration in rds mutant mice mosaic for expression of a rescue transgene.

Authors: W Kedzierski; D Bok; G H Travis
Journal: J Neurosci Date: 1998-06-01 Impact factor: 6.167

Review 3. The genomic, biochemical, and cellular responses of the retina in inherited photoreceptor degenerations and prospects for the treatment of these disorders.

Authors: Alexa N Bramall; Alan F Wright; Samuel G Jacobson; Roderick R McInnes
Journal: Annu Rev Neurosci Date: 2010 Impact factor: 12.449

4. Ciliary neurotrophic factor (CNTF) for human retinal degeneration: phase I trial of CNTF delivered by encapsulated cell intraocular implants.

Authors: Paul A Sieving; Rafael C Caruso; Weng Tao; Hanna R Coleman; Darby J S Thompson; Keri R Fullmer; Ronald A Bush
Journal: Proc Natl Acad Sci U S A Date: 2006-02-27 Impact factor: 11.205

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. Effect of gene therapy on visual function in Leber's congenital amaurosis.

Authors: James W B Bainbridge; Alexander J Smith; Susie S Barker; Scott Robbie; Robert Henderson; Kamaljit Balaggan; Ananth Viswanathan; Graham E Holder; Andrew Stockman; Nick Tyler; Simon Petersen-Jones; Shomi S Bhattacharya; Adrian J Thrasher; Fred W Fitzke; Barrie J Carter; Gary S Rubin; Anthony T Moore; Robin R Ali
Journal: N Engl J Med Date: 2008-04-27 Impact factor: 91.245

7. 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

8. Cellular interactions implicated in the mechanism of photoreceptor degeneration in transgenic mice expressing a mutant rhodopsin gene.

Authors: P C Huang; A E Gaitan; Y Hao; R M Petters; F Wong
Journal: Proc Natl Acad Sci U S A Date: 1993-09-15 Impact factor: 11.205

9. Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial.

Authors: William W Hauswirth; Tomas S Aleman; Shalesh Kaushal; Artur V Cideciyan; Sharon B Schwartz; Lili Wang; Thomas J Conlon; Sanford L Boye; Terence R Flotte; Barry J Byrne; Samuel G Jacobson
Journal: Hum Gene Ther Date: 2008-10 Impact factor: 5.695

Review 10. Loss of daylight vision in retinal degeneration: are oxidative stress and metabolic dysregulation to blame?

Authors: Claudio Punzo; Wenjun Xiong; Constance L Cepko
Journal: J Biol Chem Date: 2011-11-10 Impact factor: 5.157

21 in total

Review 1. What Is Next for Retinal Gene Therapy?

Authors: Luk H Vandenberghe
Journal: Cold Spring Harb Perspect Med Date: 2015-04-15 Impact factor: 6.915

2. Silencing of tuberin enhances photoreceptor survival and function in a preclinical model of retinitis pigmentosa (an american ophthalmological society thesis).

Authors: Stephen H Tsang; Lawrence Chan; Yi-Ting Tsai; Wen-Hsuan Wu; Chun-Wei Hsu; Jin Yang; Joaquin Tosi; Katherine J Wert; Richard J Davis; Vinit B Mahajan
Journal: Trans Am Ophthalmol Soc Date: 2014-07

Retinal gene therapy coming of age.

Review 1. Retinitis pigmentosa.

2. Non-cell-autonomous photoreceptor degeneration in rds mutant mice mosaic for expression of a rescue transgene.

Review 3. The genomic, biochemical, and cellular responses of the retina in inherited photoreceptor degenerations and prospects for the treatment of these disorders.

4. Ciliary neurotrophic factor (CNTF) for human retinal degeneration: phase I trial of CNTF delivered by encapsulated cell intraocular implants.

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

6. Effect of gene therapy on visual function in Leber's congenital amaurosis.

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

8. Cellular interactions implicated in the mechanism of photoreceptor degeneration in transgenic mice expressing a mutant rhodopsin gene.

9. Treatment of leber congenital amaurosis due to RPE65 mutations by ocular subretinal injection of adeno-associated virus gene vector: short-term results of a phase I trial.

Review 10. Loss of daylight vision in retinal degeneration: are oxidative stress and metabolic dysregulation to blame?

Review 1. What Is Next for Retinal Gene Therapy?

2. Silencing of tuberin enhances photoreceptor survival and function in a preclinical model of retinitis pigmentosa (an american ophthalmological society thesis).

Review 3. Attenuation of Inherited and Acquired Retinal Degeneration Progression with Gene-based Techniques.

4. Curing blindness: Vision quest.

Review 5. Gene therapy approaches for the treatment of retinal disorders.

6. Limited time window for retinal gene therapy in a preclinical model of ciliopathy.

7. In vivo versus ex vivo CRISPR therapies for retinal dystrophy.

Review 8. Promising and delivering gene therapies for vision loss.

9. Halting progressive neurodegeneration in advanced retinitis pigmentosa.

10. It's never too late to save a photoreceptor.