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 |
---|---|---|---|
COMPOSITIONS AND METHODS FOR IMMUNE ONCOLOGY | 2016 |
|
RU2771624C2 |
THERAPY METHODS BASED ON CAR T-CELLS WITH INCREASED EFFICIENCY | 2016 |
|
RU2788131C2 |
ANTIBODIES TO TAU AND THEIR USE | 2018 |
|
RU2787779C2 |
DOSING REGIMEN OF A WNT INHIBITOR AND AN ANTI-PD-1 ANTIBODY MOLECULE IN A COMBINATION | 2018 |
|
RU2767533C2 |
TREATING CANCER USING A CHIMERIC ANTIGENIC CLL-1 RECEPTOR | 2015 |
|
RU2741120C2 |
ANTIBODIES THAT BIND TUMOR TISSUE FOR DIAGNOSIS AND TREATMENT | 2020 |
|
RU2806915C2 |
ANTIBODIES TO ENTPD2, TYPES OF COMBINATION THERAPY AND METHODS FOR USING ANTIBODIES AND TYPES OF COMBINATION THERAPY | 2019 |
|
RU2790991C2 |
ANTIBODIES SPECIFIC FOR CD70 AND THEIR USE | 2019 |
|
RU2801093C2 |
ANTI-LAG-3 DOSING REGIMENS AND THEIR USE | 2018 |
|
RU2801208C2 |
MOLECULES OF ANTIBODIES TO CD73 AND THEIR APPLICATION WAYS | 2018 |
|
RU2791192C2 |
Authors
Dates
2024-01-30—Published
2016-12-26—Filed