A Quantitative Chloride Channel Conductance Assay for Efficacy Testing of AAV.BEST1-Reference-Cited by-同舟云学术

A Quantitative Chloride Channel Conductance Assay for Efficacy Testing of AAV.BEST1

Published:2019-04 Issue:2 Volume:30 Page:44-52
ISSN:1946-6536
Container-title:Human Gene Therapy Methods
language:en
Short-container-title:Human Gene Therapy Methods

Author:

Wood Shaun R.¹,McClements Michelle E.¹,Martinez-Fernandez de la Camara Cristina¹,Patrício Maria I.¹,Uggenti Carolina²,Sekaran Sumathi¹,Barnard Alun R.¹,Manson Forbes D.²,MacLaren Robert E.¹³⁴

Affiliation:

1. Nuffield Laboratory of Ophthalmology, Department of Clinical Neurosciences, University of Oxford, Oxford, United Kingdom.

2. Division of Evolution and Genomic Sciences, The University of Manchester, Manchester, United Kingdom.

3. National Institute for Health Research (NIHR) Oxford Biomedical Research Centre (BRC), Oxford, United Kingdom.

4. Oxford Eye Hospital, Oxford University Hospitals NHS Foundation Trust, Oxford, United Kingdom.

Publisher

Mary Ann Liebert Inc

Subject

Genetics(clinical),Pharmacology,Genetics,Applied Microbiology and Biotechnology,Molecular Medicine

Link

https://www.liebertpub.com/doi/pdf/10.1089/hgtb.2018.267

Reference30 articles.

1. The spectrum of ocular phenotypes caused by mutations in the BEST1 gene

2. Biallelic Mutation of BEST1 Causes a Distinct Retinopathy in Humans

3. Autosomal Recessive Bestrophinopathy

4. Bestrophin 1 and retinal disease

5. Bestrophin, the product of the Best vitelliform macular dystrophy gene (VMD2), localizes to the basolateral plasma membrane of the retinal pigment epithelium

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