Answer 1:
This is a great question!
You have identified a really big challenge when
it comes to using cell based therapies in
the central nervous system, especially the brain.
You've probably heard about the
"blood - brain
barrier" and how it prevents substances like
toxins from entering the brain. Now we are talking
about trying to provide new neurons to the brain
as a repair therapy. Right now, the only way we
have to do that is through an invasive
mechanism -
by delivering the cells directly into the brain,
to the location where we need the
repair/replacement to occur.
Even if we can do that, we still have a worry that
using a stem cell based therapy could result in
cells that "misbehave" - for example, will they
know when to stop dividing? If not, the patient
may develop a brain tumor. Cell based therapies -
whether or not they involve any kind of gene
editing - are still in the early days of
development. But already there are some
successes,
such as delivering retinal cells derived from stem
cells into the eyes of people with macular
degeneration (a type of age-related
blindness).
Some of those therapies are actually developed
right here at
UCSB!
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