FightSMA’s History in Gene Therapy Research

2010 FightSMA’s Science Director, Dr. Chris Lorson, applauded for his participation, leadership, and contribution as a faculty member to its 13th Annual Meeting by the American Society of Gene & Cell Therapy.
2010 FightSMA joins with Families of SMA, SMA Foundation, and MDA to sponsor a symposium on “Expanding Frontiers in Spinal Muscular Atrophy Gene Therapy” at the 13th Annual Meeting of the American Society of Gene & Cell Therapy.
2010 FightSMA partners with Gwendolyn Strong Foundation to award Dr. Brian Kaspar a $250,000 grant to support safety and toxicity studies for AAV(-9), a potential “delivery system” for gene therapy for the treatment of SMA.
2010 The journal Nature Biotechnology published article by Dr. Alex MacKenzie, Co-chair of FightSMA’s Scientific Advisory Committee, about efforts to develop a cure for SMA using gene therapy.
2009 At the FightSMA Annual Researchers Meeting, Dr. Brian Kaspar, of The Ohio State University and The Research Institute at Nationwide Children’s Hospital, updates attendees on his work with AAV and SMA mice.
2008 FightSMA joins with Families of SMA, SMA Foundation, MDA, and ALS Association to sponsor a symposium attended by approximately 400 scientists, on “Innovations in Gene Therapy for Neurodegenerative Diseases” at the 11th Annual Meeting of the American Society of Gene Therapy.
2008 At the FightSMA Annual Researchers Meeting, gene therapy strategies and the development of vectors are discussed by gene therapy experts Dr. Brian Kaspar and Dr. Nick Boulis.
2007-2009 FightSMA funds development of transgenic pig SMA model for the future development and testing of therapeutics and evaluation of toxicology, at University of Missouri by Dr. Monique Lorson.
2007 FightSMA is an official sponsor of the 10th Annual Meeting of the American Society of Gene Therapy during which members of multiple labs who have received funding from FightSMA present their research.

Tristan Coady, from the lab of FightSMA Science Director Dr. Chris Lorson, presents on topic of “SMA Based Therapeutic Trans-Splicing RNAs Repair SMN1 Deficiency”

2007 At the FightSMA Annual Researchers Meeting, a significant portion of the speaking schedule is devoted experts in the field of gene therapy, including Dr. Brian Kaspar, Dr. R. Jude Samulski, and Dr. Marco Passini.
2006-2010 FightSMA funds AAV9 gene therapy research at The University of Sheffield (United Kingdom) by Professor of Translational Neuroscience, Dr. Mimoun Azzouz, et al.
2006-2008 FightSMA funds an investigation into non-viral gene therapy research at Tor Vergata University School of Medicine (Italy) by Associate Professor of Human Genetics, Dr. Federica Sangiuolo.
2006 FightSMA sponsors a booth and reception at the 9th Annual Meeting of the American Society of Gene Therapy in order to facilitate greater communication between SMA researchers and gene therapy researchers.

Travis Baughan, from the Lab of FightSMA Science Director Dr. Chris Lorson, presents on topic of “Stimulating Full-Length SMN2 Expression by Delivering Bi-Functional RNAs Via a Viral Vector”

2006 During the FightSMA Annual Researchers Meeting, Dr. Nick Boulis, of the Cleveland Clinic, updates attendees about his lab’s work to develop gene therapy vectors and gene therapy delivery systems.
2005 FightSMA sponsors a booth and a reception at the 8th Annual Meeting of the American Society of Gene Therapy in order to raise awareness of SMA in the gene therapy community and of the exciting opportunities for gene therapy applications.
2005 During the FightSMA Annual Researchers Meeting, Dr. Nick Boulis, of the Cleveland Clinic, presents about attempts to develop a gene therapy vector and discusses the need for development of a large animal model (such as a pig) for optimum study of delivery techniques.
2003 FightSMA funds investigation of modified adenovirus and adenoviral vectors at the Gene Therapy Center at the University of Alabama-Birmingham by the Center’s Director, Dr. David Curiel.
2002-2004 FightSMA funds investigation of LentiVector® gene delivery technology at Oxford BioMedica by Dr. Nicholas Mazarakis, Dr. Mimoun Azzouz, et al.