Research Tools

Neurofibromatosis Therapeutic Acceleration Program (NTAP) supports programs that foster collaboration and encourage the open and timely sharing of results. The reagents and knowledge that are generated in our programs are made available to the neurofibromatosis community to ensure discoveries are advanced as quickly as possible.

Details about the cell lines, animal models, and other reagents that have been generated to discover and develop therapies for plexiform neurofibromas will be uploaded here as they become available.

CELL LINES:

ipNF95.11b “C”
ipnNF95.11C
ipNF95.6, 2λ
sipNF05.5
ipn02.3, 2λ
ipNF06.2A
ipNF00.6
ipn02.8

Publication: The first report of immortalization and detailed characterization of multiple human NF1 normal nerve and neurofibroma-derived Schwann cell lines (auspices of Dr. Wallace, University of Florida)

CIDr/NTAP Collaboration(1) Genetic characterization studies (Cell Line Authentication, SNP Array, Exome Sequencing, and RNA Sequencing) of NF1-/-, NF1-/+, and NF1+/+ cell-lines to be available as tool-set for community, and (2) Analyses conducted by Sage Bionetworks.

Sage AnalysisBioinformatics analyses of pNF cell lines’ high throughput screening data.

Screening (Single Agent MIPEs, combo): As a result of a collaboration between UFL, NTAP, and NCATS, a panel of cell culture systems that represented pNF complexity was used to screen new compounds (as single and combination agents) and identify novel therapeutic targets.

Validation Studies: The goal of these experiments (conducted at IU) was to validate the results of preliminary HTS at NIH/NCATS by Dr. Ferrer.

MOUSE MODELS:

DhhCre-Nf1fl/fl     

  • Uses:  Therapeutics and gene expression analysis; a key model used as part of the NFTC collaboration.
  • Source: Nancy Ratner, Ph.D. (Nancy.Ratner@cchmc.org)

Krox20;Nf1flox/−

  • Uses: Therapeutics and dosing schedules and investigating patterns of resistance;  a key model used as part of the NFTC collaboration.
  • Source: D. Wade Clapp, M.D. (dclapp@iupui.edu)

Transgenic Nf1 Nf1+/-; p53+/-   

  • Uses: Safety, Biomarker therapy; model Dr. Staedtke (one of NTAP’s Francis Collins Scholars) uses for her project evaluating Clostridium Novi for MPNST
  • Source: Karen Cichowski, Ph.D. (kcichowski@rics.bwh.harvard.edu), Tyler Jacks, Ph.D. (tjacks@mit.edu)

Nestin-CreER+; Nf1fl/fl or flox/+

  • Uses: MEK-inhibitor treatment strategy; model that Dr. Bornhorst (one of NTAP’s Francis Collins Scholars) uses in probing the timing of critical events preceding onset of OPG.
  • Source: Yuan Zhu, Ph.D. (yzhu@childrensnational.org)

Nf1-/- ;R26R-LacZ-Luciferase DNSC 

  • Uses: Cells of origin
  • Source: Lu Le, M.D., Ph.D. (Lu.Le@UTSouthwestern.edu)