Sequence specific reagents targeting CCR5 in primary hematopoietic cells
AssigneesCellectis, Albert-Ludwigs-Universität Freiburg
InventorsToni Cathomen, Tatjana Cornu, Philippe Duchateau, Claudio Mussolino, Marianna Romito, Agnes Gouble
Life of this patent
Estimating the term…
Abstract
The invention pertains to the field of cell therapy and HIV treatments. It provides with highly specific reagents for reducing or inactivating expression of CCR5 in primate and human primary cells, especially under the form of TALE-nucleases. These reagents allow the production of safer primary hematopoietic cells made resistant to HIV, stem cells or differentiated cells, for their infusion into HIV patients.
Claims24
A method for producing HIV resistant primary hematopoietic cells comprising at least the steps of:
- (i) providing a population of primary hematopoietic cells originating from a patient or a donor;
- (ii) introducing into said cells a nucleic acid encodinq a sequence specific reagent targeting at least one genomic sequence encoding the N-terminal hydrophilic external region of the CCR5 protein, wherein said specific reagent is a TALE-nuclease that binds a target sequence selected from SEQ ID NO:5 and SEQ ID NO:6;
- (iii) expressing said sequence specific reagent, so that the expression of functional CCR5 is reduced by more than 50% in the resulting population of primary cells.
A TALE-nuclease monomer designed to cleave a genomic sequence encoding the N-terminal hydrophilic external region of the CCR5 protein, wherein said monomer binds a target sequence of SEQ ID NO:5 or SEQ ID NO:6.
Dependencies are inferred from the claim text.