Accession Number:

ADA564162

Title:

Induced Pluripotent Stem Cells as Potential Therapeutic Agents in NF1

Descriptive Note:

Final rept. 1 May 2010-30 Apr 2010

Corporate Author:

INSTITUTE FOR CANCER RESEARCH PHILADELPHIA PA

Personal Author(s):

• Chernoff, Jonathan

Report Date:

2012-05-01

Pagination or Media Count:

9.0

Abstract:

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.

Descriptors:

• *CHEMOTHERAPEUTIC AGENTS
• *STEM CELLS
• BLOOD CELLS
• DEFECTS(MATERIALS)
• DRUGS
• GROWTH(PHYSIOLOGY)
• HEMATOPOIETIC CELLS
• VECTOR ANALYSIS

Subject Categories:

• Biochemistry
• Medicine and Medical Research

Distribution Statement:

APPROVED FOR PUBLIC RELEASE