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Full-Text Articles in Biomedical Engineering and Bioengineering
Nonviral Approaches For Delivery Of Crispr-Cas9 Into Hepatocytes For Treatment Of Inherited Metabolic Disease, Tanner R. Rathbone
Nonviral Approaches For Delivery Of Crispr-Cas9 Into Hepatocytes For Treatment Of Inherited Metabolic Disease, Tanner R. Rathbone
All Dissertations
Inherited metabolic diseases (IMDs) of the liver have a collective occurrence of about 1 in 800 births. Current therapies for IMDs of the liver are limited, and the only curative option for patients is orthotopic liver transplantation. Due to shortages of organ donors, immunosuppression following the transplantation, and mortality risks associated with the procedure, alternative treatment options are necessary for curing IMDs of the liver. Therapeutic gene editing has been proposed as a potential strategy for treating IMDs of the liver. Adeno-associated viral vectors (AAVs) are the most widely used delivery method for liver-targeted gene therapies. Although widely used for …