Efficacy and Safety of Retinal Gene Therapy Using Adeno-Associated Virus Vector for Patients With Choroideremia-Reference-Cited by-同舟云学术

Efficacy and Safety of Retinal Gene Therapy Using Adeno-Associated Virus Vector for Patients With Choroideremia

Published:2019-11-01 Issue:11 Volume:137 Page:1247
ISSN:2168-6165
Container-title:JAMA Ophthalmology
language:en
Short-container-title:JAMA Ophthalmol

Author:

Fischer M. Dominik¹²³⁴,Ochakovski G. Alex¹²,Beier Benjamin³,Seitz Immanuel P.¹²,Vaheb Yousof¹,Kortuem Constanze¹,Reichel Felix F. L.¹²,Kuehlewein Laura¹,Kahle Nadine A.³,Peters Tobias³,Girach Aniz⁵,Zrenner Eberhart¹²,Ueffing Marius²,MacLaren Robert E.⁴⁵⁶⁷,Bartz-Schmidt Karl Ulrich¹,Wilhelm Barbara³

Affiliation:

1. University Eye Hospital, University of Tübingen, Tübingen, Germany

2. Institute for Ophthalmic Research, Centre for Ophthalmology, University of Tübingen, Tübingen, Germany

3. STZ eyetrial at the Centre for Ophthalmology, University Hospital Tübingen, Tübingen, Tübingen, Germany

4. Nuffield Laboratory of Ophthalmology, University of Oxford, Oxford, United Kingdom

5. Nightstar Therapeutics, London, United Kingdom

6. Oxford Eye Hospital, Oxford University Hospitals National Health Service Foundation Trust, Oxford, United Kingdom

7. Moorfields Eye Hospital National Health Service Foundation Trust, London, United Kingdom

Publisher

American Medical Association (AMA)

Subject

Ophthalmology

Link

https://jamanetwork.com/journals/jamaophthalmology/articlepdf/2748910/jamaophthalmology_fischer_2019_oi_190059.pdf

Reference26 articles.

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2. Choroideremia: a review of general findings and pathogenesis.;Coussa;Ophthalmic Genet,2012

3. Remodeling of the human retina in choroideremia: rab escort protein 1 (REP-1) mutations.;Jacobson;Invest Ophthalmol Vis Sci,2006

4. Retinal degeneration in choroideremia: deficiency of rab geranylgeranyl transferase.;Seabra;Science,1993

5. Measurement and reproducibility of preserved ellipsoid zone area and preserved retinal pigment epithelium area in eyes with choroideremia.;Hariri;Am J Ophthalmol,2017

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