FIELD: genetic engineering.
SUBSTANCE: group of inventions relates to genome editing systems and methods of treating hemoglobinopathies. The following is proposed: a targeting RNA molecule for editing the BCL11a enhancer region. gRNA contains tracr and crRNA, where crRNA contains the target domain with SEQ ID NO: 253. Also the following is proposed: a nucleic acid that encodes a gRNA molecule, an expression vector, a method of changing the target sequence in a CD34+ cell, a modified CD34+ cell and compositions for use in the treatment of hemoglobinopathy.
EFFECT: inventions lead to high induction of fetal hemoglobin (HbF) and induction of HbF-positive cells, which contributes to the treatment of hemoglobinopathy.
60 cl, 49 dwg, 34 tbl, 10 ex
| Title | Year | Author | Number |
|---|---|---|---|
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|
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| ANTIBODIES SPECIFIC FOR CD70 AND THEIR USE | 2019 |
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| ANTI-LAG-3 DOSING REGIMENS AND THEIR USE | 2018 |
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RU2801208C2 |
