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Thesis

Development of CRISPR-Cas genome and epigenome engineering tools towards retinal degenerations

Abstract:

Inherited retinal diseases (IRDs) are a diverse family of disorders caused by genetic mutations that lead to dysfunction and death of cells in the outer retina. Together, they are the most common cause of untreatable blindness in the working age population. While gene supplementation therapies using adeno-associated viral (AAV) vectors have shown restoration of visual function in IRDs, they are limited to the treatment of recessively inherited diseases caused by small genes. The engineerin...

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Division:
MSD
Department:
Clinical Neurosciences
Role:
Author

Contributors

Role:
Supervisor
Institution:
University of Oxford
Division:
MSD
Department:
Clinical Neurosciences
Role:
Supervisor
ORCID:
0000-0002-2941-4464
More from this funder
Name:
Rhodes Scholarships
Funder identifier:
http://dx.doi.org/10.13039/501100000697
Type of award:
DPhil
Level of award:
Doctoral
Awarding institution:
University of Oxford
DOI:
Language:
English
Keywords:
Subjects:
Deposit date:
2023-02-22

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