Abstract

Public Health Relevance

Lay summary: This proposal would test a wholly new approach to a possible genetic therapy for chromosomal trisomy, for which gene therapy is not currently considered feasible. We would assess the feasibility of silencing the extra chr 21 in human DS cells and in a mouse DS model using the system nature uses to silence one X chr. in females.

  Funding Agency

Agency
National Institute of Health (NIH)
Institute
National Institute of General Medical Sciences (NIGMS)
Type
Research Project (R01)
Project #
5R01GM085548-02
Application #
7666833
Study Section
Special Emphasis Panel (ZGM1-GDB-7 (EU))
Program Officer
Carter, Anthony D
Project Start
2008-08-01
Project End
2012-07-31
Budget Start
2009-08-01
Budget End
2010-07-31
Support Year
2
Fiscal Year
2009
Total Cost
$328,083
Indirect Cost

  Institution

Name
University of Massachusetts Medical School Worcester
Department
Anatomy/Cell Biology
Type
Schools of Medicine
DUNS #
603847393
City
Worcester
State
MA
Country
United States
Zip Code
01655

  Publications

Jiang, Jun; Jing, Yuanchun; Cost, Gregory J et al. (2013) Translating dosage compensation to trisomy 21. Nature 500:296-300
Hall, L L; Lawrence, J B (2010) XIST RNA and architecture of the inactive X chromosome: implications for the repeat genome. Cold Spring Harb Symp Quant Biol 75:345-56