Media coverage
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Media coverage
Title Brain Foundation Research Gift Degree of recognition National Media name/outlet Brain Foundation of Australia Media type Web Country/Territory Australia Date 21/10/19 Description Our goal is to develop new ways
to treat a blood vessel disorder
that occurs in the brain called
arteriovenous malformations, or
AVMs. These tangled collections
of abnormal blood vessels can
form in the brain during early
development. They are highly
prone to rupturing, leading to
release of blood into the brain,
causing a type of stroke. This
form of stroke occurs primarily in
children and young adults, rather
than as a result of aging, so has
a significant impact on affected
individuals and their families.
Despite current approaches, one-
third of these vulnerable young
patients lack safe treatment
options and remain susceptible
to stroke. To fill this gap, we aim
to develop a vascular targeting
approach to AVM treatment. This
involves delivering a drug through
the bloodstream to induce
localised clotting and closure of
the diseased AVM vessels. Key
to this approach is 1) identifying
molecular targets unique to the
surface of the diseased blood
vessels but absent from normal,
healthy blood vessels; and 2)
developing complementary
targeting molecules that
recognise and bind these targets
to deliver vessel-occluding drugs
specifically to the AVMs. The recent
discovery that more than half of all
AVMs are caused by mutations in
a family of genes called RAS, for
the first time provides a defined
molecular cell type for AVMs that
can be easily modelled for study
in the laboratory. Our study aims
to develop an AVM cell culture
model in the laboratory that
expresses the RAS mutation
and then use it to generate novel
targeting molecules (called DNA
aptamers) that can specifically
recognise and bind RAS-induced
molecular targets on the surface
of these mutant cells. These novel
targeting molecules could then be
used to deliver vessel-blocking
drugs with high specificity to
AVM vessels providing a safe and
effective new treatment approach
for AVM patients.URL https://brainfoundation.org.au/research-grants/2019/cerebral-diseases-15/ Persons Lucinda McRobb, Marcus Stoodley