Hi Everyone,
I'm new here...my son has a very rare genetic disorder called Pitt Hopkins...it results in severe physical and intellectual disabilities as well as lack of speech. It has been put on the autism spectrum because of the sensory and communication issues...but is quite a bit different...In fact the kids tend to be very social.
That said, I'm wondering if anyone can give me any advice/thoughts re: stem cells for single gene disorders...Is this being done? Or given that it is a genetic disorder would the autologous cells also be compromised and therefore ineffective for stem cell treatment? Any advice or knowledge would be much a appreciated! THank you so much! Audrey
I'm new here...my son has a very rare genetic disorder called Pitt Hopkins...it results in severe physical and intellectual disabilities as well as lack of speech. It has been put on the autism spectrum because of the sensory and communication issues...but is quite a bit different...In fact the kids tend to be very social.
That said, I'm wondering if anyone can give me any advice/thoughts re: stem cells for single gene disorders...Is this being done? Or given that it is a genetic disorder would the autologous cells also be compromised and therefore ineffective for stem cell treatment? Any advice or knowledge would be much a appreciated! THank you so much! Audrey