Spinal Muscular ATrophy (SMA)

 

Spinal muscular Atrophy affects approximately 1 in 6000 births.


There is Currently no Cure for the disease.


We are working to identify novel approaches and therapeutics for The treatment of SMA 

Tetracyclines That Promote SMN2 Exon 7 Splicing as Therapeutics for Spinal Muscular Atrophy
Michelle L. Hastings, Joel Berniac, Ying Hsiu Liu, Paul Abato, Francine M. Jodelka, Lea Barthel, Sujatha Kumar, Caroline Dudley, Mark Nelson, Kelley Larson, Jason Edmonds, Todd Bowser, Michael Draper, Paul Higgins, Adrian Krainer. 
Science Translational Medicine, 4 November 2009: 5ra12http://stm.sciencemag.org/content/1/5.toc

SUPPORT

A Feedback Loop Regulates Splicing of the Spinal Muscular Atrophy-Modifying Gene, SMN2
Francine M. Jodelka, Alison D. Ebert, Dominik M. Duelli, Michelle L. Hastings
Human Molecular Genetics, 5 December, 2010, 19: 4906-4917http://stm.sciencemag.org/content/1/5.toc

Targeting SR proteins improves SMN expression in spinal muscular atrophy cells.

Wee CD, Havens MA, Jodelka FM, Hastings ML.

PLoS One. 2014 Dec 15;9(12):e115205. doi: 10.1371/journal.pone.0115205.

A Feedback Loop Regulates Splicing of the Spinal Muscular Atrophy-Modifying Gene, SMN2
Francine M. Jodelka, Alison D. Ebert, Dominik M. Duelli, Michelle L. Hastings
Human Molecular Genetics, 5 December, 2010, 19: 4906-4917http://stm.sciencemag.org/content/1/5.toc