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1.
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2.
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Phenylketonuria PKU is an autosomal recessive inherited metabolic disorder that can cause neurocognitive dysfunction and mental retardation if dietary treatment is not in [...]
2014-06-01 |
Thesis |
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3.
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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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The main focus of this dissertation work was to advance gene therapy research for phenylketonuria (PKU). PKU is an inborn error of metabolism characterized by phenylalani [...]
2020 |
Dissertation |
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6.
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PKU requires lifelong management, but the childhood PKU diet does not accommodate for post-adolescent patient variations, including: (1) the diet's effect on blood phenyl [...]
2020 |
Abstract |
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8.
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The long-chain polyunsaturated fatty acids (LCPUFA) docosahexaenoic acid (DHA; C22:6n-3) and arachidonic acid (ARA; C20:4n-6) have important physiological implications an [...]
2007-05-01 |
Thesis |
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9.
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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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10.
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