Possible therapies

Possible Therapies
It was speculated that it may become possible to repair the disease locus by selected and targeted addition of 3.3 kb repeats to the disease locus on chromosome 4. Such targeted gene therapy might prevent development of the FSHD phenotype, but the practical feasibility of such an approach is as yet unknown.

>> This we cannot do because of a lack of knowledge.<<

Another approach to explore is the modification of cultured FSHD regenerative muscle cells that would reverse their higher sensitivity to oxidative stress. Such cultured cells, with better ability to respond to oxidative stress, might then be used for treatment of patients. It would be valuable to prevent the reintroduced cells from again developing increased sensitivity.

>> This we can do. This is the approach I am taking.<<

http://accessible.ninds.nih.gov/news_and_events/proceedings/fshmdconference.h...

Blessings,
-Donna