New MiniPromoter Ple345 (NEFL) Drives Strong and Specific Expression in Retinal Ganglion Cells of Mouse and Primate Retina-Reference-Cited by-同舟云学术

New MiniPromoter Ple345 (NEFL) Drives Strong and Specific Expression in Retinal Ganglion Cells of Mouse and Primate Retina

Published:2019-03 Issue:3 Volume:30 Page:257-272
ISSN:1043-0342
Container-title:Human Gene Therapy
language:en
Short-container-title:Human Gene Therapy

Author:

Simpson Elizabeth M.¹²³⁴,Korecki Andrea J.¹,Fornes Oriol¹²,McGill Trevor J.⁵⁶,Cueva-Vargas Jorge Luis⁷,Agostinone Jessica⁷,Farkas Rachelle A.¹,Hickmott Jack W.¹²,Lam Siu Ling¹,Mathelier Anthony¹²,Renner Lauren M.⁵,Stoddard Jonathan⁵,Zhou Michelle¹,Di Polo Adriana⁷,Neuringer Martha⁵⁶,Wasserman Wyeth W.¹²

Affiliation:

1. Centre for Molecular Medicine and Therapeutics at BC Children's Hospital, University of British Columbia, Vancouver, British Columbia, Canada.

2. Department of Medical Genetics, University of British Columbia, Vancouver, British Columbia, Canada.

3. Department of Ophthalmology and Visual Sciences, University of British Columbia, Vancouver, British Columbia, Canada.

4. Department of Psychiatry, University of British Columbia, Vancouver, British Columbia, Canada.

5. Division of Neuroscience, Oregon National Primate Research Center, Oregon Health and Science University, Portland, Oregon.

6. Department of Ophthalmology, Casey Eye Institute, Oregon Health and Science University, Portland, Oregon.

7. Department of Neuroscience and Centre de Recherche du Centre Hospitalier de l'Université de Montréal, Université de Montréal, Montréal, Québec, Canada.

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

https://www.liebertpub.com/doi/pdf/10.1089/hum.2018.118

Reference53 articles.

1. Gene Therapy 2017: Progress and Future Directions

2. Molecular genetics and emerging therapies for retinitis pigmentosa: Basic research and clinical perspectives

3. Optimization of Retinal Gene Therapy for X-Linked Retinitis Pigmentosa Due to RPGR Mutations

4. Nonclinical Safety Evaluation of scAAV8- RLBP1 for Treatment of RLBP1 Retinitis Pigmentosa

5. Photoreceptor rescue by an abbreviated human RPGR gene in a murine model of X-linked retinitis pigmentosa

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