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0:00
Ladies and gentlemen, my
name is Christian Buchholz.
I'm working at the
Paul-Ehrlich-Institut,
where I'm supervising
a research group that
is working on
vector engineering.
In this presentation,
I will show you, in particular,
how engineered vector
particles specific for
particular cell types
can be used for
in vivo CAR T cell generation.
0:27
The presentation
is starting with
an introduction to gene
therapy and CAR T cells.
We will then go into
the concept of receptor-targeted
viral vectors.
I will then switch
to, as announced,
in vivo CAR T cell generation,
and we will then have
a closer look at the
so-called DARPins,
which is a very key
polypeptide used to
generate the targeted
viral vectors.
Finally, we will have
a look at a new generation
of viral vectors,
the so-called
DART-AAV vectors for
their application
in immunotherapy.
1:08
The gene therapy
field is experiencing
a huge boom during
the recent years,
and we have meanwhile
close to 20 products that
have been authorized for
their use on the market
in the US and the EU.
We can, in general,
distinguish between
gene therapy products that
are applied ex vivo and
those applied in vivo.
Ex vivo means that the
relevant cells are
removed from the
patient and then
genetically modified
in cell culture,
before being reinfused
again into the patient.
Among this type of product,
we have the CAR T cells,
as you can see on
the bottom left,
there are meanwhile
six different types of
CAR T cells that have
reached the market.
On the other hand,
we have genetically modified
hematopoietic stem cells,
and this is actually
the key strategy to
cure genetic diseases,
for example, the severe
combined immunodeficiencies
or sickle cell anemia,
which is another example of
the genetic diseases that are
targeted by this ex vivo
gene transfer strategy.
As I mentioned,
the other possibility is
in vivo gene transfer,
where you have
vectors directly injected
into the patients and then
the genetic modification
of cells occurs within
the patient's body.
As you can see on
the bottom right,
this is the AAV vectors
in particular that
are used for this purpose,
and examples here are
also genetic diseases,
especially hemophilia,
where we have meanwhile
four different products that
have reached the market.
