The FDA approved Human Clinical Trials for Gene Therapy!!!!!
The press release from Nationwide Children’s Hospital is below:
What this means in layman’s terms…..they utilize a simple Adeno Associated Virus derived from non-human primates. This virus is cleaned out of all the bad things and used as a “shuttle”. The SMN 1 protein which is the missing protein in SMA patients is inserted into the shuttle virus. This virus has evolved and is one of the few viruses that can efficiently cross the blood brain barrier and target motor neurons. The cells that die in SMA patients due to a lack of the SMN 1 protein. The virus gets inside the MN and leaves it’s payload for the MN the SMN 1 protein. It is a 1 time delivery delivered through IV to the bloodstream. The protein self replicates inside the cell. The therapy is the only one to show a full reversal in the severe SMA mouse model when delivered early. This therapy has also rescued an SMA pig model and has shown to target motor neurons in Non-human primates very effectively. It is the by far the greatest pre-clinical data ever seen for SMA and now it will be tried in humans.”
This is a HUGE answer to many of your prayers. We have been praying daily for this research to move forward and it happened in part by the donations that our family received from all of you and passed on to Sophia’s Cure Foundation to be used to research. Thank you again and continue to pray that these trials will show great results