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Targeting bcl11a enhancer functional regions for fetal hemoglobin reinduction

Application No.

PCT/US2016/031224

Broad Case No.

BI-2015/097

List of inventors -

Feng Zhang, Neville Sanjana, Stuart Orkin, Ophir Shalem, Jason Wright, Daniel Bauer

Abstract

Provided herein are nucleic acid molecules that target the BCL11A enhancer functional regions, compositions comprising the nucleic acid molecules and methods for increasing fetal hemoglobin levels in a cell by disrupting BCL11A expression at the genomic level. Also provided herein are methods and compositions relating to the treatment of hemoglobinopathies by reinduction of fetal hemoglobin levels. In particular, the nucleic acid molecules target the +62, +58, and/or the +55 enhancer functional regions.

Date PUBLISHED

  • 11/17/2016

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