0:00
So hello, everybody.
This so the first recording in
this series of gene therapy.
I am in the Luigi Naldini.
I am the director of the
San Raffaele-Telethon
Institute for Gene
Therapy in Milan, Italy.
And I will be discussing,
in this first lecture,
general features of gene
therapy and vectors.
0:22
In the first slide, we are
mentioning the key strategies
in gene transfer and gene therapy.
Gene addition, which
is the insertion
of new genetic information in a cell
to replace a malfunctioning gene
and replacing its function, or
to instruct a novel function
to that cell-- for
instance, to make that cell
resistant to an infectious
agent or to help
that cell better fight the cancer.
These are the most common strategies
in gene transfer and therapy today.
There are, however,
additional strategies, which
are gene subtraction--
in which case,
we aim to disrupt or inhibit an
endogenous gene to essentially
prevent its expression, again, to
enable maybe resistance to a virus
or to fight a dominant
oncogenic mutation.
And gene editing, which
is a more advanced
of gene therapy in which, rather
than adding or subtracting genes,
we are actually rewriting the
genetic sequencing in the cell,
most often to correct directly in situ, a genetic inherited
mutation, or to insert novel
sequence at this specific site.
And gene editing can be performed
at the RNA level, in which case
we would modify RNA transcript,
either by, for instance,
forcing axon skipping
or trans-splicing.
Or in a more stable at the DNA
level by either disrupting genes
or forcing homologous
recombination through the use
of artificial nucleuses.
And all of these strategies
will be, of course,
discussed in detail
in selected talks.
