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[BMD RES 5HA] - Final Exam Guide - Comprehensive Notes fot the exam (34 pages long!)


Department
Biomedical Research
Course Code
BMD RES 5HA
Professor
Robert Romero
Study Guide
Final

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UCLA
BMD RES 5HA
FINAL EXAM
STUDY GUIDE

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Duchenne Muscular Dystrophy (DMD)
X-linked disease caused by mutations in the dystrophin
gene
The dystrophin-glycoprotein complex (DGC) protects
the sarcolemma during muscle contraction
Loss of DGC leads to decreased cell adhesion in DMD
Sarcospan is the smallest core component of the DGC
and UGC
DMD patient doesn't have expression of Dystrophin and
SSPN
The role of sarcospan is unclear
In terms of dystrophy, it acts like a "molecular
glue"
Mediate interactions between transmembrane
components of the dystrophin-glycoprotein
mSSPN overexpression increases abundance of UGC
and DGC components at the sarcolemma
mSSPN overexpression reduces central nucleation in 6
week mdx quadriceps
mSSPN overexpression improves muscle membrane
stability in mdx mice
High expression of mouse sarcospan increases forelimb
grip strength in mdx mice
mSSPN reduces mdx susceptibility to eccentric
contraction damage
Transgenic expression of mouse sarcospan reduces
dystrophic pathology of mdx diaphragm
SSPN expression improves pulmonary function in mdx
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mice
DMD symptoms are more mild in mice than in humans
Whole body plethysmography
Summary: sarcospan can enhance membrane stability in
DMD and leads to reduced muscle degeneration
Ongoing sarcospan research
How does SSPN enhance UGC/DGC components
at the membrane?
Can we treat DMD with AAV-mediated SSPN
expression (gene therapy)?
Can SSPN enhance other therapeutic approaches in
DMD?
AAV delivery of dystrophin or utrophin
Pharmacologic upregulation of utrophin
"exon skipping" through antisense
oligonucleotide targeting of dystrophin
Muscle has high regenerating capacities
Grip strength is not too hard to look at with mice
Mice are born with both mdx and the trans gene
Preventing most of the damage, but not halting it at a
certain point
Mdx mice don't have the same progression as humans
6 week is about teenager, not quite yet an adult for a mouse
6 months mouse is old
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