Homology directed repair compositions for the treatment of hemoglobinopathies
AssigneeSeattle Children's Hospital
InventorsAndrew Scharenberg, Kyle Jacoby, Hans-Peter Kiem, David J. Rawlings, Christopher Lux, Sowmya Pattabhi, Olivier M. Humbert
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Abstract
The present disclosure provides improved compositions for the homology directed repair of the human globin locus for the prevention, treatment, or amelioration of at least one symptom of a hemoglobinopathy.
Claims18
A method for increasing gamma globin expression in a hematopoietic stem or progenitor cell comprising introducing into the cell one or more engineered nucleases that cleave a target site set forth in SEQ ID NO:
- 9 and
- a DNA donor repair template having the sequence as set forth in SEQ ID NOs:
- 17, 18, 19, 20, or 21,
- whereby the DNA donor repair template is inserted into the cell genome by homology directed repair at a double strand break introduced by the one or more engineered nucleases.
A method for increasing gamma globin expression in a hematopoietic stem or progenitor cell comprising introducing into the cell
- one or more engineered nucleases that cleave a target site set forth in SEQ ID NO:
- 9 and
- a DNA donor repair template having at least 99% sequence identity to the sequence as set forth in SEQ ID NOs:
- 17, 18, 19, 20, or 21,
- whereby the DNA donor repair template is inserted into the cell genome by homology directed repair at a double strand break introduced by the one or more engineered nucleases.
A method for increasing gamma globin expression in a hematopoietic stem or progenitor cell comprising introducing into the cell
- one or more engineered nucleases that cleave a target site set forth in SEQ ID NO:
- 9 and
- a DNA donor repair template having at least 98% sequence identity to the sequence as set forth in SEQ ID NOs:
- 17, 18, 19, 20, or 21,
- whereby the DNA donor repair template is inserted into the cell genome by homology directed repair at a double strand break introduced by the one or more engineered nucleases.
Dependencies are inferred from the claim text.