Compositions comprising an rna guide targeting bcl11a and uses thereof
AssigneeArbor Biotechnologies, Inc.
InventorsQuinton Norman Wessells, Jeffrey Raymond Haswell, Tia Marie Ditommaso, Noah Michael Jakimo
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Abstract
The present invention relates to compositions comprising RNA guides targeting BCL11A, processes for characterizing the compositions, cells comprising the compositions, and methods of using the compositions.
Claims92
A composition comprising an RNA guide, wherein the RNA guide comprises (i) a spacer sequence that is substantially complementary to a target sequence within a BCL11A gene and (ii) a direct repeat sequence;
- wherein the target sequence is adjacent to a protospacer adjacent motif (PAM) comprising the sequence 5′-NTTN-3′.
An RNA guide comprising (i) a spacer sequence that is substantially complementary to a target sequence within a BCL11A gene and (ii) a direct repeat sequence.
The composition, RNA guide, nucleic acid, vector, cell, kit or method of any one of the previous claims, wherein the composition, RNA guide, nucleic acid, vector, cell, kit or method disrupts a GATAA motif of an enhancer region of the BCL11A gene.
The composition, cell, kit or method of any one of the previous claims, wherein the composition, cell, kit or method comprises at least two RNA guides targeting a GATAA motif of an enhancer region of the BCL11A gene.
The composition, RNA guide, nucleic acid, vector, cell, kit or method of any one of the previous claims, wherein the RNA guide consists of the sequence of:
- (SEQ ID NO:
- 2677)
- AGAAAUCCGUCUUUCAUUGACGGGAAGCUAGUCUAGUGCAAGC;
- (SEQ ID NO:
- 2678)
- AGAAAUCCGUCUUUCAUUGACGGCUGGAGCCUGUGAUAAAAGC;
- and/or
- (SEQ ID NO:
- 2679)
- AGAAAUCCGUCUUUCAUUGACGGUACCCCACCCACGCCCCCAC.
The composition, RNA guide, nucleic acid, vector, cell, kit or method of any one of the previous claims, wherein the RNA guide does not consist of the sequence of:
- (SEQ ID NO:
- 2677)
- AGAAAUCCGUCUUUCAUUGACGGGAAGCUAGUCUAGUGCAAGC;
- (SEQ ID NO:
- 2678)
- AGAAAUCCGUCUUUCAUUGACGGCUGGAGCCUGUGAUAAAAGC;
- and/or
- (SEQ ID NO:
- 2679)
- AGAAAUCCGUCUUUCAUUGACGGUACCCCACCCACGCCCCCAC.
Dependencies are inferred from the claim text.