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.
|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|