Liver-directed gene therapy

Published on February 23, 2022   34 min

A selection of talks on Pharmaceutical Sciences

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Hello. Welcome to my talk on Liver-Directed Gene Therapy. My name is Nicola Brunetti-Pierri and I work at the Telethon Institute of Genetics and Medicine and at Federico II University in Naples, Italy.
The focus of this talk is on gene therapy- that is the treatment of diseases using nucleic acids, either DNA or RNA. Viruses are typically used as a tool for transferring therapeutic genes because viruses have the natural ability to transfer genetic material to the infected cells. The viruses are modified in the laboratory to preserve their natural capacity for transferring genetic material, but the harmful genes of the viruses are deleted. The viral vector typically binds to a receptor on the plasma membrane, is internalized and coated in the cytoplasm, and then the viral genome migrates into the nuclei where it can stay as an episome, or it can integrate into the chromosomal DNA. Either as an episomal or as an integrated vector, the viral vector genome can then produce the therapeutic gene product.
Liver transplantation is used for the treatment of several liver metabolic diseases. Liver transplantation is performed with the goal of correcting a single gene defect by replacing thousands of genes that are from a donor organ. The goal of gene therapy instead, is to target correction of the disease-causing gene defect.