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1.
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The goal of this dissertation is to improve our understanding of the factors governing genetic repair to facilitate whole gene insertion into the genome of mature organis [...]
2025-02-07 |
Dissertation |
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2.
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Current treatments for FTD mitigate some behavioral changes, but there are no therapies that prevent or slow the progression of the disease. Profiling the epigenetic chan [...]
2025-01-15 |
Dissertation |
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3.
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The goal of this dissertation is to improve understanding and treatment for the chorioretinopathy seen in long-chain 3-hydroxyacyl-CoA dehydrogenase deficiency (LCHADD) p [...]
2024-06-13 |
Dissertation |
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4.
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Liver-directed gene and cell therapies represent a promising therapeutic strategy for many genetic disorders. However, they are hampered by inefficiency. The problem of i [...]
2023-12-15 |
Dissertation |
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