Literature DB >> 32556084

Quantitative Autofluorescence Following Gene Therapy With Voretigene Neparvovec.

Sarah R Levi1, Jin Kyun Oh1, Jose Ronaldo Lima de Carvalho1,2, Vinit B Mahajan3, Stephen H Tsang1,4, Janet R Sparrow1,4.   

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Year:  2020        PMID： 32556084      PMCID： PMC9578385          DOI： 10.1001/jamaophthalmol.2020.2018

Source DB:  PubMed          Journal:  JAMA Ophthalmol        ISSN： 2168-6165            Impact factor:   8.253


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  6 in total

1.  Efficacy, Safety, and Durability of Voretigene Neparvovec-rzyl in RPE65 Mutation-Associated Inherited Retinal Dystrophy: Results of Phase 1 and 3 Trials.

Authors:  Albert M Maguire; Stephen Russell; Jennifer A Wellman; Daniel C Chung; Zi-Fan Yu; Amy Tillman; Janet Wittes; Julie Pappas; Okan Elci; Kathleen A Marshall; Sarah McCague; Hannah Reichert; Maria Davis; Francesca Simonelli; Bart P Leroy; J Fraser Wright; Katherine A High; Jean Bennett
Journal:  Ophthalmology       Date:  2019-06-22       Impact factor: 12.079

Review 2.  Lessons learned from quantitative fundus autofluorescence.

Authors:  Janet R Sparrow; Tobias Duncker; Kaspar Schuerch; Maarjaliis Paavo; Jose Ronaldo Lima de Carvalho
Journal:  Prog Retin Eye Res       Date:  2019-08-28       Impact factor: 21.198

3.  Rpe65 is necessary for production of 11-cis-vitamin A in the retinal visual cycle.

Authors:  T M Redmond; S Yu; E Lee; D Bok; D Hamasaki; N Chen; P Goletz; J X Ma; R K Crouch; K Pfeifer
Journal:  Nat Genet       Date:  1998-12       Impact factor: 38.330

4.  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

5.  Lack of fundus autofluorescence to 488 nanometers from childhood on in patients with early-onset severe retinal dystrophy associated with mutations in RPE65.

Authors:  Birgit Lorenz; Bettina Wabbels; Erika Wegscheider; Christian P Hamel; Wolfgang Drexler; Markus N Preising
Journal:  Ophthalmology       Date:  2004-08       Impact factor: 12.079

6.  Sustained Rescue of Rod Function and Probable Non-Cell-Autonomous Rescue of Cones after Gene Therapy.

Authors:  Karen Sophia Park; Jose Ronaldo Lima de Carvalho; Stephen H Tsang
Journal:  Ophthalmology       Date:  2019-09       Impact factor: 14.277
  6 in total
  2 in total

1.  A novel phenotype in a family with autosomal dominant retinal dystrophy due to c.1430A > G in retinoid isomerohydrolase (RPE65) and c.37C > T in bestrophin 1 (BEST1).

Authors:  Juanita Pappalardo; Rachael C Heath Jeffery; Jennifer A Thompson; Enid Chelva; Quang Pham; Ian J Constable; Terri L McLaren; Tina M Lamey; John N De Roach; Fred K Chen
Journal:  Doc Ophthalmol       Date:  2021-01-29       Impact factor: 2.379

Review 2.  Clinical Perspective: Treating RPE65-Associated Retinal Dystrophy.

Authors:  Albert M Maguire; Jean Bennett; Elena M Aleman; Bart P Leroy; Tomas S Aleman
Journal:  Mol Ther       Date:  2020-12-03       Impact factor: 11.454
  2 in total

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