SMA
Nov. 17, 2016
Live long and prosper: healthy mitochondria, healthy motor neurons?
Chris Lorson (front) and Mark Hannink (back) collaborate to study the role of mitochondria in motor neuron health, particularly in relation to spinal muscular atrophy, a neuromuscular disorder | photo by Jen Lu, Bond LSC Chris Lorson, a professor of veterinary pathobiology, and Mark Hannink, a professor of biochemistry, want to find a new way to help motor neurons live a long and healthy life. Their question: what’s the relationship between motor neuron sruvival and a cellular component called mitochondria? The two researchers at the Bond Life Sciences Center were awarded preliminary funding from the Bond…
July 21, 2016
Finding hope by fixing a gene
Lorson lab publishes research on a new therapeutic path to help treat spinal muscular atrophy By Phillip Sitter | MU Bond Life Sciences Center Erkan Osman shows iImages of neuro-muscular junctions. Osman, a post-doctoral fellow in Chris Lorson’s lab, co-authored research in the journal Molecular Therapy that details work in binding a synthetic nucleic acid to a normally useless motor neuron backup gene to help treat spinal muscular atrophy. | photo by Phillip Sitter, Bond LSC Imagine you are forced to jump out of an airplane. Luckily, you find a parachute that even…
July 16, 2014
Researchers flex new muscle in SMA drug development
By Paige Blankenbuehler Lauren and Claire Gibbs share contagious laughter, ambition and a charismatic sarcasm. Both are honor students at Shawnee Mission East High School in a Kansas City suburb. They also share a neuromuscular disease called spinal muscular atrophy (SMA), designated as an “orphan disease” because it affects fewer than 200,000 people in the U.S. However, the landscape for individuals with SMA is quickly changing with the development of new drugs. More than 7 million people in the United States are carriers (approximately 1 in 40) of the so-called “rare” neurodegenerative disease, SMA. Lauren,17 (left) and Claire, 16 (right),…