Journal article icon

Journal article

Initial results from a first-in-human gene therapy trial on X-linked retinitis pigmentosa caused by mutations in RPGR

Abstract:

Retinal gene therapy has shown great promise in treating retinitis pigmentosa (RP), a primary photoreceptor degeneration that leads to severe sight loss in young people. In the present study, we report the first-in-human phase 1/2, dose-escalation clinical trial for X-linked RP caused by mutations in the RP GTPase regulator (RPGR) gene in 18 patients over up to 6 months of follow-up (https://clinicaltrials.gov/: NCT03116113). The primary outcome of the study was safety, and secondary o...

Expand abstract
Publication status:
Published
Peer review status:
Peer reviewed

Actions


Access Document


Files:
Publisher copy:
10.1038/s41591-020-0763-1

Authors


More by this author
Role:
Author
ORCID:
0000-0002-9956-6412
More by this author
Institution:
University of Oxford
Division:
MSD
Sub department:
Clinical Neurosciences
Role:
Author
ORCID:
0000-0003-0065-9131
More by this author
Institution:
University of Oxford
Division:
MSD
Sub department:
Clinical Neurosciences
Role:
Author
ORCID:
0000-0002-6612-6162
Expand authors...
Department of Health More from this funder
More from this funder
Grant:
CL-2014-13-003
ICA-CDRF-2016-02-002
NF-SI-0515-10047
NIHR201338
Publisher:
Springer Nature Publisher's website
Journal:
Nature Medicine Journal website
Volume:
26
Issue:
3
Pages:
354-359
Place of publication:
United States
Publication date:
2020-02-24
Acceptance date:
2020-01-10
DOI:
EISSN:
1546-170X
ISSN:
1078-8956
Pmid:
32094925
Language:
English
Keywords:
Pubs id:
1088911
Local pid:
pubs:1088911
Deposit date:
2021-08-22

Terms of use


Views and Downloads






If you are the owner of this record, you can report an update to it here: Report update to this record

TO TOP