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Hi, I'm Jonathan Chernoff of the Fox Chase Cancer Center, and I've been interested in
NF 1 for some time now. I've recently come up with an idea for an unusual treatment possibility
in NF 1, which I'll describe. So NF 1 has some unusual properties that make unusual
treatment possible, and that is that NF 1, unlike many other diseases, has a contribution
from the bone marrow which is absolutely essential for the progression of the disease. So based
on that idea we've been thinking about using induced progenitor cells (or stem cells) as
a possible treatment. And with some support from the Children's Tumor Foundation we've
started a project where we are using a mouse model of NF 1, taking out skin cells from
these mice, converting them into stem cells, fixing the gene--the NF 1 gene--and then putting
them back by transplant into the mice with the idea that we'll prevent these mice from
ever getting the NF 1. If this would work, it would open up a potential therapeutic in
people with the disease. This is quite an unusual idea, and we are very grateful for
the Children's Tumor Foundation for funding it because we didn't think that this sort
of high-risk project could get conventional funding, and indeed, with the Children's Tumor
Foundation's help and some pilot data, we've now been able to obtain funding from the Department
of Defense to enlarge the project. So we are grateful for the support and especially for
support for unusual ideas, and we hope that his will lead to a new therapeutic.