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 [...]

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 [...]

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 [...]

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 [...]

Ovarian teratoma, comprising cystic mature teratomas and malignant immature teratomas, is a common cancer of the female reproductive tract that tends to occur in young wo [...]

Normal placentation is essential for the health and well-being of both the mother and fetus throughout pregnancy. In humans, defective placental development contributes t [...]

This dissertation describes the development of the TRADE system for directed evolution of the AAV capsid, its successful application to identify the HN1 variant, a novel [...]

Single-cell methods have proven to be a powerful tool in the interrogation of complex biology. One such biological system in which single-cell methods have been paramount [...]

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 [...]

During healthy development and aging, somatic mutations aggregate in tissues, lending different mutational profiles to cells within these tissues. Similarly, developmenta [...]