Induced Pluripotent Stem Cells as Potential Therapeutic Agents in NF1
Abstract
In this project, we are seeking to use induced pluripotent stem (iPS) cell technology as a potential therapy in neurofibromatosis 1 (NF1). In the first year, 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 have created a targeting "gene-repair" vector to replace the defective Nf1 allele in these iPS cells. Thus, we are on schedule for this project, having achieved our aims in the expected time frame.
Document Details
- Document Type
- Technical Report
- Publication Date
- May 01, 2011
- Accession Number
- ADA547230
Entities
People
- Jonathan Chernoff