0:00
This is Dave Williams,
I'm chief of hematology/oncology
at Boston Children's Hospital
and Dana Farber Cancer Institute.
And I'm going to be
talking today about gene
transfer and gene therapy.
0:14
So what I would like
to cover quickly today
is outlined in the first slide.
We'll talk a little bit about
the definition of gene therapy,
the requirements for
success of gene therapy,
in particular focusing on blood
diseases as a model system,
talk just briefly about applicable
vector systems that can be used
in blood diseases including
gamma retroviruses,
lentiviruses, foamy
and alpha retroviruses,
then briefly review experience
from clinical trials,
including the adenosine
deaminase or ADA-SCID
trial, the X-SCID trial, CGD, WAS,
CCALD trial, and beta-thalassemia,
and finally, talk about
insertional mutagenesis
as it relates to these
clinical trials and experience
with leukemias,
and end with a couple slides on
prospects and new approaches,
including new cell
targets and gene editing.
1:11
OK, so the first slide is just
a general introduction to gene
therapy by way of definition.
So we think of gene
therapy as introducing
new genetic material into
the cells of an organism
for therapeutic purposes.
There's essentially two broad
concepts or types: germline,
in which the DNA is
introduced in the germ cells
and therefore the new material
can be passed into the gene pool,
and somatic, in which the
gene sequences are introduced
into specialized somatic
cells, and therefore
the genetic material
is really limited
to the individual recipient.
And of course, the former germline
gene therapy is not practiced;
in fact is banned in most countries,
and the latter is what
we'll be focusing on today.
