Literature DB >> 29570997

Gene Therapy for Retinal Degeneration.

Rajendra S Apte1.   

Abstract

Biallelic mutations in the RPE65 gene are associated with inherited retinal degenerations/dystrophies (IRD) and disrupt the visual cycle, leading to loss of vision. A new adenoviral vector-based gene therapy surgically delivered to retinal cells provides normal human RPE65 protein that can restore the visual cycle and some vision. To view this Bench to Bedside, open or download the PDF.
Copyright © 2018. Published by Elsevier Inc.

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Year:  2018        PMID： 29570997     DOI： 10.1016/j.cell.2018.03.021

Source DB:  PubMed          Journal:  Cell        ISSN： 0092-8674            Impact factor:   41.582


  23 in total

1.  Challenges to Routine Genetic Testing for Inherited Retinal Dystrophies.

Authors:  Albert S Li; Donna MacKay; Howard Chen; Rithwick Rajagopal; Rajendra S Apte
Journal:  Ophthalmology       Date:  2019-04-24       Impact factor: 12.079

Review 2.  VEGF in Signaling and Disease: Beyond Discovery and Development.

Authors:  Rajendra S Apte; Daniel S Chen; Napoleone Ferrara
Journal:  Cell       Date:  2019-03-07       Impact factor: 41.582

Review 3.  Retinal stem cell transplantation: Balancing safety and potential.

Authors:  Mandeep S Singh; Susanna S Park; Thomas A Albini; M Valeria Canto-Soler; Henry Klassen; Robert E MacLaren; Masayo Takahashi; Aaron Nagiel; Steven D Schwartz; Kapil Bharti
Journal:  Prog Retin Eye Res       Date:  2019-09-05       Impact factor: 21.198

4.  A CEP290 C-Terminal Domain Complements the Mutant CEP290 of Rd16 Mice In Trans and Rescues Retinal Degeneration.

Authors:  Suddhasil Mookherjee; Holly Yu Chen; Kevin Isgrig; Wenhan Yu; Suja Hiriyanna; Rivka Levron; Tiansen Li; Peter Colosi; Wade Chien; Anand Swaroop; Zhijian Wu
Journal:  Cell Rep       Date:  2018-10-16       Impact factor: 9.423

5.  Multisensory perception in Argus II retinal prosthesis patients: Leveraging auditory-visual mappings to enhance prosthesis outcomes.

Authors:  Noelle R B Stiles; Vivek R Patel; James D Weiland
Journal:  Vision Res       Date:  2021-02-17       Impact factor: 1.886

6.  Optoretinography of individual human cone photoreceptors.

Authors:  Robert F Cooper; David H Brainard; Jessica I W Morgan
Journal:  Opt Express       Date:  2020-12-21       Impact factor: 3.894

7.  Detection and validation of novel mutations in MERTK in a simplex case of retinal degeneration using WGS and hiPSC-RPEs model.

Authors:  Pooja Biswas; Shyamanga Borooah; Hiroko Matsui; Marina Voronchikhina; Jason Zhou; Qais Zawaydeh; Pongali B Raghavendra; Henry Ferreyra; S Amer Riazuddin; Karl Wahlin; Kelly A Frazer; Radha Ayyagari
Journal:  Hum Mutat       Date:  2020-12-13       Impact factor: 4.700

8.  Seven novel variants expand the spectrum of RPE65-related Leber congenital amaurosis in the Chinese population.

Authors:  Zilin Zhong; Feng Rong; Yinghui Dai; Alakezi Yibulayin; Lin Zeng; Jian Liao; Liefeng Wang; Zhihua Huang; Zhenping Zhou; Jianjun Chen
Journal:  Mol Vis       Date:  2019-03-18       Impact factor: 2.367

9.  Mining Disaggregase Sequence Space to Safely Counter TDP-43, FUS, and α-Synuclein Proteotoxicity.

Authors:  Amber Tariq; JiaBei Lin; Meredith E Jackrel; Christina D Hesketh; Peter J Carman; Korrie L Mack; Rachel Weitzman; Craig Gambogi; Oscar A Hernandez Murillo; Elizabeth A Sweeny; Esin Gurpinar; Adam L Yokom; Stephanie N Gates; Keolamau Yee; Saurabh Sudesh; Jacob Stillman; Alexandra N Rizo; Daniel R Southworth; James Shorter
Journal:  Cell Rep       Date:  2019-08-20       Impact factor: 9.423

Review 10.  The cGMP Pathway and Inherited Photoreceptor Degeneration: Targets, Compounds, and Biomarkers.

Authors:  Arianna Tolone; Soumaya Belhadj; Andreas Rentsch; Frank Schwede; François Paquet-Durand
Journal:  Genes (Basel)       Date:  2019-06-14       Impact factor: 4.096
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