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Targeting Bcl11a distal regulatory elements for fetal hemoglobin reinduction

A DNA-targeting, artificial blood technology, applied in the direction of hemoglobin/myoglobin, peptide/protein components, blood diseases, etc., can solve problems that are not well understood, etc.

Active Publication Date: 2018-09-25
CHILDRENS MEDICAL CENT CORP
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  • Abstract
  • Description
  • Claims
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AI Technical Summary

Problems solved by technology

Unfortunately, the in vivo molecular mechanisms underlying globin turnover are not well understood

Method used

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  • Targeting Bcl11a distal regulatory elements for fetal hemoglobin reinduction
  • Targeting Bcl11a distal regulatory elements for fetal hemoglobin reinduction
  • Targeting Bcl11a distal regulatory elements for fetal hemoglobin reinduction

Examples

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Embodiment

[0349] The present inventors discovered and characterized regulatory elements of the BCL11A gene that are critical for BCL11A gene expression in cells of the erythroid lineage. Common genetic variants in these sequences were associated with fetal hemoglobin levels and the severity of beta-globin disturbances. These sequences contain distal regulatory elements with enhancer chromatin marks, accessible chromatin, activated histone marks, and occupied by erythroid transcription factors. These elements interact with the BCL11A promoter and promote gene expression in erythroid cells, but not in other lineages (eg, B lymphocytes) that express BCL11A. These regulatory elements can be focused for therapeutic purposes to achieve inhibition of BCL11A and re-induction of fetal hemoglobin. This can be achieved through mechanisms not limited to genome editing, nucleic acid binding or protein binding, and epigenetic modification. Advantages of the method include: disruption of physiologic...

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Abstract

Provided herein are methods and compositions for increasing fetal hemoglobin levels in cells by disrupting expression of BCL11A at the genomic level. Also provided herein are methods and compositions directed to the treatment of hemoglobinopathies by reinduction of fetal hemoglobin levels.

Description

[0001] Cross References to Related Applications [0002] Pursuant to 35 U.S.C. §119(e), this application claims U.S. Provisional Application No. 61 / 730,323 filed November 27, 2012, U.S. Provisional Application No. 61 / 730,369 filed November 27, 2012, March 11, 2013 Priority to U.S. Provisional Application No. 61 / 776,144, filed October 10, 2013, and U.S. Provisional Application No. 61 / 889,174, filed October 10, 2013, the contents of each of which are hereby incorporated by reference in their entirety. [0003] governmental support [0004] This invention was made with Government support under Grant No. 5RO1HL032259 awarded by the National Institutes of Health. The US Government has certain rights in this invention. Background technique [0005] Normal adult hemoglobin contains four globins, two alpha proteins and two beta proteins. During fetal development in mammals, especially humans, the fetus produces fetal hemoglobin, which contains two gamma-globins instead of two beta-...

Claims

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Application Information

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Patent Type & Authority Patents(China)
IPC IPC(8): C12N5/10C12N5/0789C12N15/09
CPCC07K14/805C12N15/102C12N15/63C07K14/4702A61K38/465C12N9/22C12N15/01A61P7/00A61P7/06A61K35/12
Inventor 斯图尔特·H·奥尔金丹尼尔·E·鲍尔徐剑
Owner CHILDRENS MEDICAL CENT CORP
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