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US 2021/0230638 A1Published application
Systems and methods for the treatment of hemoglobinopathies
AssigneeEditas Medicine, Inc.
InventorsEdouard AUPEPIN DE LAMOTHE-DREUZY, KaiHsin CHANG, Minerva Elaine SANCHEZ, Jack HEATH
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Abstract
Genome editing systems, guide RNAs, and CRISPR-mediated methods are provided for altering portions of the HBG1 and HBG2 loci in cells and increasing expression of fetal hemoglobin.
Claims21
1-43. (canceled)
A synthetic guide RNA (gRNA) comprising a targeting domain complementary to a sequence located within one or more regions selected from the group consisting of Chr 11 (NC_000011.10):
- 5,249,198-5,249,362 (Region 3);
- 5,249,591-5,249,712 (Region 4);
- 5,250,464-5,250,549 (Region 10);
- 5,250,594-5,250,735 (Region 11);
- 5,253,425-5,254,121 (Region 12);
- 5,254,122-5,254,306 (Region 13);
- 5,254,511-5,254,648 (Region 14);
- 5,255,255-5,255,292 (Region 20);
- and 5,255,518-5,255,641 (Region 21).
6 more elements
A genome editing system, comprising:
- an RNA-guided nuclease;
- and
- at least one guide RNA (gRNA) comprising a targeting domain complementary to a sequence within one or more regions selected from the group consisting of Chr 11 (NC_000011.10):
- 5,249,198-5,249,362 (Region 3);
- 5,249,591-5,249,712 (Region 4);
- 5,250,464-5,250,549 (Region 10);
- 5,250,594-5,250,735 (Region 11);
- 5,253,425-5,254,121 (Region 12);
- 5,254,122-5,254,306 (Region 13);
- 5,254,511-5,254,648 (Region 14);
- 5,255,255-5,255,292 (Region 20);
- and 5,255,518-5,255,641 (Region 21).
9 more elements
A CRISPR-mediated method of altering a cell, comprising:
- introducing a first DNA single strand break (SSB) or double strand break (DSB) within a region selected from the group consisting of Chr 11 (NC_000011.10):
- 5,249,198-5,249,362 (Region 3);
- 5,249,591-5,249,712 (Region 4);
- 5,250,464-5,250,549 (Region 10);
- 5,250,594-5,250,735 (Region 11);
- 5,253,425-5,254,121 (Region 12);
- 5,254,122-5,254,306 (Region 13);
- 5,254,511-5,254,648 (Region 14);
- 5,255,255-5,255,292 (Region 20);
- and 5,255,518-5,255,641 (Region 21).
7 more elements
Dependencies are inferred from the claim text.