Lentiviral vectors for site-specific gene insertion
AssigneeCity of Hope
InventorsJiing-Kuan Yee, Gilles Michel
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Abstract
Murine leukemia virus (MLV) and lentivirus vectors have been used previously to deliver genes to hematopoietic stem cells (HSCs) in human gene therapy trials. However, these vectors integrate randomly into the host genome, leading to disruption or inactivation of vital host genes. The present invention discloses a novel lentiviral vector system that overcomes this problem by integrating into a host genome in a site-specific manner.
Claims23
A lentiviral-based targeting vector for site-specific insertion of an exogenous polynucleotide sequence into a host genome comprising the exogenous polynucleotide sequence, a lentiviral polynucleotide sequence, and a fusion protein capable of stimulating homologous recombination.
A method for inserting an exogenous polynucleotide sequence into a host genome in a site-specific manner, comprising transducing a host cell with a lentiviral-based targeting vector comprising the exogenous polynucleotide sequence, a lentiviral polynucleotide sequence, and a fusion protein capable of stimulating homologous recombination.
A fusion protein comprising the HIV preintegration complex protein Vpr attached to the amino or carboxy terminus of the Cre protein or a Cre variant.
A fusion protein comprising the HIV preintegration complex protein Vpr attached to Scel.
A fusion protein comprising the HIV preintegration complex protein Vpr attached to a chimeric nuclease, wherein said chimeric nuclease comprises a DNA binding domain attached to an endonuclease domain.
Dependencies are inferred from the claim text.