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0:00
Hello.
And welcome to the next installment
of the Henry Stewart Recorded
Lecture Series on the
science of gene therapy.
My name is John Gray.
Today I'll be providing a
general overview of the science
of manufacturing viral
gene therapy vectors.
0:17
The manufacturer of viral
gene therapy vectors
is essentially a co-opting of
the natural virus life cycle
to selectively harness the natural
power of viruses to deliver genes
to cells while avoiding the
pathological consequences
of a natural viral infection.
Virologists typically study viruses
in the context of a life cycle
that begins when a viral
particle infects a naive cell.
This converts the naive cell
into a virally infected cell,
and as natural viruses contain
all the genetic information
necessary to instruct the
cell to replicate the virus,
this effectively creates
a virus-producing cell.
Because this cycle begins
with the infection event,
virologists typically describe
this infection process
as the early phase, and the
process whereby viral particles are
produced from a virally
infected cell as the late phase.
1:12
Manufacturing of viral
gene therapy vectors
is therefore an attempt to mimic the
late phase of the viral life cycle
in a clean room environment in order
to make a product which can be used
later to perform the
early phase by delivering
genetic cargo to a patient's cells.
For an injectable vector,
viral vector particles
are generated from vector
producer cells in a clean room
and are the final
vialed drug product.