Critical to the evaluation of potential therapeutics (pharmacological,
gene or cell therapies) in mouse models of muscular dystrophy are sensitive
and repeatable physiological assessments of muscle function. Therefore, we
are proposing to create a research core which performs ex vivo, in situ, and
whole animal assessments of muscle integrity and function. Three of the
projects (Projects 1-3) proposed in this grant contain components that
require muscle physiological measurements. (Project 4 relies on the outcome
of these tests for pre-clinical justification.) The Physiological Assessment
Core will meet the needs of the projects within this grant, as well as the
needs of the muscular dystrophy research community. In addition, the core
will maintain in house colonies of mouse models of DMD, LGMD, and myotonic
dystrophy in order to support studies where long-term testing is necessary.
The resources of this core are contained within Dr.
Sweeney's laboratory, which includes all instrumentation for muscle
physiological measurements, as well as specialized equipment for
hindlimb suspension experiments, documented free wheel running, and
treadmill running. Dr. Barton is a muscle physiologist with
extensive experience evaluating muscle function in mice. Monica
Zadel is an expert at performing muscle mechanical experiments,
while Pedro Acosta maintains our breeding colonies and will monitor
hindlimb suspension and free wheel running experiments. We believe
that this Core will provide a valuable resource that will enable the
entire muscular dystrophy research community to utilize these assays
to assess the potential benefits of a large number of approaches to
the treatment of different forms of muscular dystrophy.