Preclinical Dose-Escalation Study of Intravitreal AAV-RS1 Gene Therapy in a Mouse Model of X-linked Retinoschisis: Dose-Dependent Expression and Improved Retinal Structure and Function-Reference-Cited by-同舟云学术

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Preclinical Dose-Escalation Study of Intravitreal AAV-RS1 Gene Therapy in a Mouse Model of X-linked Retinoschisis: Dose-Dependent Expression and Improved Retinal Structure and Function

Published:2016-05 Issue:5 Volume:27 Page:376-389
ISSN:1043-0342
Container-title:Human Gene Therapy
language:en
Short-container-title:Human Gene Therapy

Author:

Bush Ronald A.¹,Zeng Yong¹,Colosi Peter²,Kjellstrom Sten¹³,Hiriyanna Suja²,Vijayasarathy Camasamudram¹,Santos Maria²,Li Jinbo¹,Wu Zhijian²,Sieving Paul A.¹²

Affiliation:

1. National Institute on Deafness and Other Communication Disorders, National Institutes of Health, Bethesda, Maryland.

2. National Eye Institute, National Institutes of Health, Bethesda, Maryland.

3. Department of Ophthalmology, Lund University, Lund, Sweden.

Publisher

Mary Ann Liebert Inc

Subject

Genetics,Molecular Biology,Molecular Medicine

Link

http://www.liebertpub.com/doi/pdf/10.1089/hum.2015.142

Reference42 articles.

1. Effect of Gene Therapy on Visual Function in Leber's Congenital Amaurosis

2. Reversal of Blindness in Animal Models of Leber Congenital Amaurosis Using Optimized AAV2-mediated Gene Transfer

3. Human gene therapy for RPE65 isomerase deficiency activates the retinoid cycle of vision but with slow rod kinetics

4. HumanRPE65Gene Therapy for Leber Congenital Amaurosis: Persistence of Early Visual Improvements and Safety at 1 Year

5. Vision 1 Year after Gene Therapy for Leber's Congenital Amaurosis

Cited by 61 articles. 订阅此论文施引文献订阅此论文施引文献，注册后可以免费订阅5篇论文的施引文献，订阅后可以查看论文全部施引文献

1. Retinal organoids with X-linked retinoschisis RS1 (E72K) mutation exhibit a photoreceptor developmental delay and are rescued by gene augmentation therapy;Stem Cell Research & Therapy;2024-05-31

2. OCT Intensity of the Region between Outer Retina Band 2 and Band 3 as a Biomarker for Retinal Degeneration and Therapy;Bioengineering;2024-05-01

3. Intravitreal injection of new adeno-associated viral vector: Enhancing retinoschisin 1 gene transduction in a mouse model of X-linked retinoschisis;Biochemistry and Biophysics Reports;2024-03

4. The dose-response relationship of subretinal gene therapy with rAAV2tYF-CB-hRS1 in a mouse model of X-linked retinoschisis;Frontiers in Medicine;2024-02-13

5. The Road towards Gene Therapy for X-Linked Juvenile Retinoschisis: A Systematic Review of Preclinical Gene Therapy in Cell-Based and Rodent Models of XLRS;International Journal of Molecular Sciences;2024-01-19

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