Methods and compositions for treatment of a genetic condition
First Claim
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1. An isolated, non-naturally occurring fusion protein comprising a transcription activator like effector (TALE) DNA binding domain that binds to a target site in an endogenous BCL11A gene comprising a nucleotide sequence selected from the group consisting of SEQ ID NO:
- 236, 238, 240 and 242, and a nuclease cleavage domain, wherein the fusion protein cleaves the endogenous BCL11A gene, wherein the TALE domain comprises a repeat variable diresidue (RVD) sequence in order presented and selected from a single row of table 1C.
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Abstract
Methods and compositions for a genetic disease are provided.
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Citations
11 Claims
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1. An isolated, non-naturally occurring fusion protein comprising a transcription activator like effector (TALE) DNA binding domain that binds to a target site in an endogenous BCL11A gene comprising a nucleotide sequence selected from the group consisting of SEQ ID NO:
- 236, 238, 240 and 242, and a nuclease cleavage domain, wherein the fusion protein cleaves the endogenous BCL11A gene, wherein the TALE domain comprises a repeat variable diresidue (RVD) sequence in order presented and selected from a single row of table 1C.
- View Dependent Claims (2, 3, 4, 5, 6, 7, 8, 9, 10, 11)
Specification