好色先生

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Abstract Details

Evaluating the Therapeutic Potential of Geranylgeraniol (GGOH) in an iPSC-Derived Duchenne Muscular Dystrophy Disease Model
Child Neurology and Developmental Neurology
P3 - Poster Session 3 (5:00 PM-6:00 PM)
8-001
To determine whether geranylgeraniol (GGOH) treatment of isogenic induced pluripotent stem cell (iPSC)-derived Duchenne muscular dystrophy (DMD) and control myoblasts promotes rescue of myotube fusion and downstream cellular processes in the DMD cells.
GGOH is a metabolite in the mevalonate pathway that contributes to the synthesis of cholesterol and other non-sterol isoprenoids. Previous studies have shown that GGOH treatment in C2C12 cells promotes myogenic differentiation while suppressing skeletal muscleatrophy. GGOH also reduces statin-induced damage in cultured mouse myotubes and zebrafish muscle. As it currently stands, GGOH is taken as a dietary supplement by some individuals taking statins. Given its ability to mitigate statin-induced muscle damage, GGOH may have relevance in other muscle diseases. Prior work suggests that the mevalonate pathway may harbor therapeutic targets for DMD.
To explore the potential of GGOH as a therapeutic agent in DMD, isogenic iPSC-derived Duchenne muscular dystrophy (DMD) and control myoblasts were cultured and treated with a gradient GGOH doses during differentiation. Myotube fusion indices were quantified, and downstream molecular characteristics, including expression of myogenic and atrophy related genes, were assessed.
Preliminary findings suggest that GGOH may have some therapeutic effects.

We anticipate that our ongoing studies will clarify the therapeutic potential of GGOH in DMD, setting the stage for further studies including human clinical trials.
Authors/Disclosures
Johnnie Turner
PRESENTER 		The institution of Mr. Turner has received research support from 好色先生.
MEKALA GUNASEKARAN (UNIVERSITY OF MINNESOTA) MEKALA GUNASEKARAN has nothing to disclose.
Sree L. Venigalla, PhD Dr. Venigalla has nothing to disclose.
Hannah Littel Hannah Littel has nothing to disclose.
Christina A. Pacak, PhD (University of Minnesota) Dr. Pacak has received personal compensation in the range of $0-$499 for serving as a Consultant for LifeSciences Consultants. An immediate family member of Dr. Pacak has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Neurogene. An immediate family member of Dr. Pacak has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Lupin. An immediate family member of Dr. Pacak has received personal compensation in the range of $500-$4,999 for serving as a Consultant for ITF Therapeutics. An immediate family member of Dr. Pacak has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for MLAB Biosciences. An immediate family member of Dr. Pacak has received personal compensation in the range of $10,000-$49,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Wiley. An immediate family member of Dr. Pacak has received personal compensation in the range of $10,000-$49,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for 好色先生. The institution of Dr. Pacak has received research support from Sarepta Therapeutics. The institution of Dr. Pacak has received research support from Regenerative Medicine MN. The institution of Dr. Pacak has received research support from Greg Marzolf Jr Foundation. The institution of Dr. Pacak has received research support from NIH. The institution of Dr. Pacak has received research support from Viljem Julijan Association for Children with Rare Diseases . The institution of an immediate family member of Dr. Pacak has received research support from Xtraordinary Joy Foundation. The institution of an immediate family member of Dr. Pacak has received research support from Centers for Disease Control and Prevention. The institution of an immediate family member of Dr. Pacak has received research support from National Institutes of Health. The institution of an immediate family member of Dr. Pacak has received research support from Food and Drug Administration. The institution of an immediate family member of Dr. Pacak has received research support from National Initiative for Cockayne Syndrome. The institution of an immediate family member of Dr. Pacak has received research support from Greg Marzolf Jr. Foundation. The institution of an immediate family member of Dr. Pacak has received research support from Sarepta Therapeutics. The institution of an immediate family member of Dr. Pacak has received research support from Duchenne UK - PPMD. The institution of Dr. Pacak has received research support from MN Partnership . Dr. Pacak has received intellectual property interests from a discovery or technology relating to health care. Dr. Pacak has received publishing royalties from a publication relating to health care. An immediate family member of Dr. Pacak has a non-compensated relationship as a Medical Advisory Board with Speak Foundation that is relevant to AAN interests or activities. An immediate family member of Dr. Pacak has a non-compensated relationship as a President (including President Elect and Past President) with Child Neurology Society that is relevant to AAN interests or activities. An immediate family member of Dr. Pacak has a non-compensated relationship as a Board Member with Child Neurology Foundation that is relevant to AAN interests or activities.
Peter B. Kang, MD, FAAN (University of Minnesota Medical School) Dr. Kang has received personal compensation in the range of $5,000-$9,999 for serving as a Consultant for Neurogene. Dr. Kang has received personal compensation in the range of $500-$4,999 for serving as a Consultant for Lupin. Dr. Kang has received personal compensation in the range of $500-$4,999 for serving as a Consultant for ITF Therapeutics (Italfarmaco). An immediate family member of Dr. Kang has received personal compensation in the range of $0-$499 for serving as a Consultant for Life Sciences Consultants. Dr. Kang has received personal compensation in the range of $500-$4,999 for serving on a Scientific Advisory or Data Safety Monitoring board for MLAB Biosciences. Dr. Kang has received personal compensation in the range of $10,000-$49,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for Wiley. Dr. Kang has received personal compensation in the range of $10,000-$49,999 for serving as an Editor, Associate Editor, or Editorial Advisory Board Member for 好色先生. The institution of Dr. Kang has received research support from Xtraordinary Joy Foundation. The institution of an immediate family member of Dr. Kang has received research support from Sarepta Therapeutics. The institution of Dr. Kang has received research support from Centers for Disease Control and Prevention (CDC). The institution of Dr. Kang has received research support from National Institutes of Health (NIH). The institution of Dr. Kang has received research support from Food and Drug Administration. The institution of Dr. Kang has received research support from National Initiative for Cockayne Syndrome. The institution of Dr. Kang has received research support from Greg Marzolf Jr Foundation. The institution of Dr. Kang has received research support from Sarepta Therapeutics. The institution of Dr. Kang has received research support from Duchenne UK - PPMD. The institution of an immediate family member of Dr. Kang has received research support from Regenerative Medicine MN. The institution of an immediate family member of Dr. Kang has received research support from Greg Marzolf Jr Foundation. The institution of an immediate family member of Dr. Kang has received research support from NIH. The institution of an immediate family member of Dr. Kang has received research support from Viljem Julijan Association for Children with Rare Disease. The institution of Dr. Kang has received research support from Minnesota Partnership. Dr. Kang has received intellectual property interests from a discovery or technology relating to health care. An immediate family member of Dr. Kang has received intellectual property interests from a discovery or technology relating to health care. Dr. Kang has received publishing royalties from a publication relating to health care. Dr. Kang has a non-compensated relationship as a Medical Advisory Board with Speak Foundation that is relevant to AAN interests or activities. Dr. Kang has a non-compensated relationship as a President (Including President-Elect and Past President) with Child Neurology Society that is relevant to AAN interests or activities. Dr. Kang has a non-compensated relationship as a Board Member with Child Neurology Foundation that is relevant to AAN interests or activities.