Journal article icon

Journal article

An AAV dual vector strategy ameliorates the Stargardt phenotype in adult Abca4−/− mice

Abstract:

The recent approval in the United States of the first adeno-associated viral (AAV) vector for the treatment of an inherited retinal degeneration validates this approach for the treatment of many other diseases. A major limiting factor continues to be the size restriction of the AAV transgene at under 5 kb. Stargardt disease is the most prevalent form of recessively inherited blindness and is caused by mutations in ABCA4, the gene that codes for ATP-binding cassette transporter protein family ...

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

Actions


Access Document


Publisher copy:
10.1089/hum.2018.156

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:
Clinical Neurosciences
More by this author
Institution:
University of Oxford
Division:
Medical Sciences Division
Department:
Clinical Neurosciences
Expand authors...
More from this funder
Funding agency for:
Maclaren, RE
More from this funder
Grant:
DPFS/DCS grant MR/K007629/1 (M.E.M. and R.E.M.)
Publisher:
Mary Ann Liebert Publisher's website
Journal:
Human Gene Therapy Journal website
Volume:
30
Issue:
5
Pages:
590-600
Publication date:
2018-10-31
Acceptance date:
2018-10-30
DOI:
EISSN:
1557-7422
ISSN:
1043-0342
Pubs id:
pubs:938372
URN:
uri:559dbd26-ca13-4ff5-b39b-c6525fca72c9
UUID:
uuid:559dbd26-ca13-4ff5-b39b-c6525fca72c9
Local pid:
pubs:938372

Terms of use


Metrics



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

TO TOP