$200K For SMA—Phase Two | Funding the pre-clinical sma model
On the heels of the success of the "$200K For SMA -- Phase One" campaign, in March 2011 theGSF launched "$200K For SMA -- Phase Two" to fund the first ever SMA pre-clinical animal model. theGSF once again enthusiastically partnered with FightSMA and their esteemed scientific advisory board to fund groundbreaking research that will impact many facets of SMA research, including gene therapy, for years to come.
So, what will be funded through the $200K For SMA -- Phase Two commitment? >> We'll be funding the development of the first large SMA pre-clinical animal model -- the first ever in the world! Like the coveted SMA mouse model advancement years ago, the pre-clinical large animal model is expected to become the standard by which future treatments will be studied and tested and is creating great excitement and enthusiasm among researchers and drug companies globally.
Why is the pre-clinical animal model needed? >>
- It should more closely mimic the human condition
- It will be closer to humans in size, physiology, metabolism, cardiac output, lung capacity, nervous system and immune system
- Its intent is to expand the therapeutic window in order to identify the most effective therapeutics
- It will provide a valuable resource for translational studies of two promising SMA therapeutic strategies: 1) gene therapy to replace SMN and 2) oligonucleotide delivery to increase SMN2 exon 7 inclusion
- It will facilitate the analysis of delivery methods, SMN expression, dosing, sustainability and immune response—all critical components for effective “bench to beside” (translational) applications