Induced Pluripotent Stem Cells as Potential Therapeutic Agents in NF1.

摘要

In this project, we are seeking to use induced pluripotent stem (iPS) cell technology as a potential therapy in NF1. In the first years, we have successfully produced iPS cells from fobroblasts from Nf1 +/-mice and characterized the properties of these cells, which include growth in clusters, expression of stem cell markers, normal karyotype, and the ability to form teratomas in mice. We also created a targeting 'gene-repair' vector and have used this vector to to replace the defective Nf1 allele in these iPS cells. We then showed that we could differentiate iPS cells into Schwann-like cells in vitro, enabling drug sensitivity studies.