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The biological activity of AAV vectors for choroideremia gene therapy can be measured by in vitro prenylation of RAB6A

Abstract:

Choroideremia (CHM) is a rare, X-linked recessive retinal dystrophy caused by mutations in the CHM gene. CHM is ubiquitously expressed in human cells and encodes Rab escort protein 1 (REP1). REP1 plays a key role in intracellular trafficking through the prenylation of Rab GTPases, a reaction that can be reproduced in vitro. With recent advances in adeno-associated virus (AAV) gene therapy for CHM showing gene replacement to be a promising approach, an assay to assess the biological activity o...

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Publication status:
Published
Peer review status:
Peer reviewed
Version:
Publisher's version

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Publisher copy:
10.1016/j.omtm.2018.03.009

Authors


More by this author
Institution:
University of Oxford
Division:
Medical Sciences Division
Department:
Clinical Neurosciences
More by this author
Institution:
University of Oxford
Division:
Medical Sciences Division
Department:
NDORMS; KIR
More by this author
Institution:
University of Oxford
Division:
Medical Sciences Division
Department:
NDORMS; KIR
Nightstar Therapeutics More from this funder
Royal College of Surgeons of Edinburgh More from this funder
National Institute for Health Research More from this funder
Publisher:
Elsevier Publisher's website
Journal:
Molecular Therapy: Methods and Clinical Development Journal website
Volume:
9
Pages:
288-295
Publication date:
2018-03-28
Acceptance date:
2018-03-25
DOI:
EISSN:
2329-0501
Pubs id:
pubs:846495
URN:
uri:bd3fbdc4-aad4-44d9-8752-e33af53db18d
UUID:
uuid:bd3fbdc4-aad4-44d9-8752-e33af53db18d
Local pid:
pubs:846495

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