Thesis
Developing a gene therapy for Stargardt Disease
- Abstract:
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Stargardt disease was first described by Stargardt in Bonn in 1909 and is the most common recessively inherited macular degeneration, affecting 1:8,000-10,000. It results in progressive vision loss and legal blindness by the fourth to seventh decade of life, therefore, it is highly clinically relevant. Stargardt disease is caused by mutations in the ABCA4 gene, which encodes the ATP-binding Cassette protein -4 (ABCA4), a transport protein essential to the visual cycle which is expressed in ph...
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- Files:
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(Preview, Dissemination version, pdf, 22.0MB, Terms of use)
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Authors
Contributors
+ MacLaren, R
- Institution:
- University of Oxford
- Division:
- MSD
- Department:
- Clinical Neurosciences
- Role:
- Supervisor
+ McClements, M
- Institution:
- University of Oxford
- Division:
- MSD
- Department:
- Clinical Neurosciences
- Role:
- Supervisor
+ Macular Society
More from this funder
- Funder identifier:
- https://ror.org/02j172648
- Grant:
- GR599
- Programme:
- PhD Studentship - Development of CRISPR gene therapy for Stargardt Disease - Jointly funded by RetinaUK and Macular Society
- DOI:
- Type of award:
- DPhil
- Level of award:
- Doctoral
- Awarding institution:
- University of Oxford
- Language:
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English
- Keywords:
- Subjects:
- Deposit date:
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2026-02-19
- ARK identifier:
Terms of use
- Copyright holder:
- Elena Piotter
- Copyright date:
- 2025
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