0:00
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.
0:16
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.
1:17
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.
