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Thesis

Gene therapy for surfactant protein B deficiency using recombinant AAV vectors

Abstract:

Gene therapies are being developed for rare genetic diseases and offer hope for conditions with few treatment options. Surfactant protein B (SP-B) deficiency is a rare genetic disease of the lung causing severe respiratory distress in neonates and is fatal within the first months of life. Recombinant adeno-associated virus (rAAV) is being investigated for delivery of functional SP-B to the lungs of SP-B deficient neonates, to produce surfactant for secretion onto the lung surface to increase ...

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Institution:
University of Oxford
Division:
MSD
Department:
RDM
Sub department:
RDM Clinical Laboratory Sciences
Research group:
Gene Medicine Group
Oxford college:
St Cross College
Role:
Author
ORCID:
0000-0001-6435-7584

Contributors

Institution:
University of Oxford
Division:
MSD
Department:
RDM
Sub department:
RDM Clinical Laboratory Sciences
Research group:
Gene Medince Group
Role:
Supervisor
ORCID:
0000-0002-5264-054X
Institution:
University of Oxford
Division:
MSD
Department:
RDM
Sub department:
RDM Clinical Laboratory Sciences
Research group:
Gene Medicine Group
Role:
Supervisor
ORCID:
0000-0002-8877-4005
Institution:
University of Oxford
Role:
Examiner
ORCID:
0000-0003-4508-4802
Institution:
University College London
Role:
Examiner
More from this funder
Programme:
PhD Scholarship
Funding agency for:
Meyer-Berg, H
More from this funder
Programme:
MRC-DTP
Funding agency for:
Meyer-Berg, H
Type of award:
DPhil
Level of award:
Doctoral
Awarding institution:
University of Oxford

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