2023-24 LLL Impact Report - Flipbook - Seite 21
2023
JOINT RESEARCH
GRANT RECIPIENT
Director Dr. Christine Vande Velde. “We are
hopeful that exploring this understudied mechanism will inform on motor neuron homeostasis
and that these models will be valuable to future
development of targeted therapeutics,” Vande
Velde also stated.
Mohassel, an associate professor of
neurology at the Johns Hopkins University School
of Medicine, specializes in myopathies, muscular
dystrophies, and other hereditary neuromuscular
disorders and is the co-director of the Johns
Hopkins Myositis Center.
“Funding from the Packard Center and
the Live Like Lou Foundation will enable us to
create new iPSC and mouse models of a novel
genetic form of ALS due to excess sphingolipid
synthesis,” noted Mohassel. “We plan to make
these models available to all investigators who
are interested in studying them to understand
the underlying mechanisms of this disease and
develop therapies. We are grateful for this
opportunity and look forward to sharing our
progress in the near future.”
DR. PAYAM
MOHASSEL
Recent work in his lab
led to the discovery of
a new genetic form of
ALS that stems from
excessive activity of an
enzyme called serine
palmitoyltransferase.
This study seeks to create cellular and animal
models of this new form of ALS to enable future
studies to help better understand and to test
candidate therapies.
“Our investment in emerging scientists brings
unique and impactful discovery to ALS research,”
shared Live Like Lou Foundation Executive
Director Wendy Faust. “We are honored to
partner with Packard to advance early-stage
researchers in their work,” Faust 昀椀nished.
This grant will generate new human neuronal
cell models and humanized mouse models in
which to study pathogenic mutations in SPTLC1,
an essential component of the sphingolipid
metabolism pathway. Sphingolipids are
specialized lipids that participate in cellular
signaling that have been implicated in neuronal
homeostasis.
“We are excited to collaborate with the Live
Like Lou Foundation to jointly fund this important
research,” noted Packard Center Scienti昀椀c
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ROBERT PACKARD CENTER
FOR ALS RESEARCH
The Robert Packard Center for ALS Research at Johns Hopkins
is an international collaborative research group of committed
scientists who tackle key questions related to the cause and
treatment of ALS. To support their studies, the Center provides
expertise, advanced technology, and a highly collaborative spirit.
The core of the Center’s mission is to 昀椀nd real therapies and
ultimately a cure for ALS. The hope is in the science.
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