Retroviral construct harboring a let-7 insensitive nucleic acid encoding hmga2 and methods of use thereof
AssigneeSt. Jude Children's Research Hospital
InventorsSheng Zhou, Brian Sorrentino, Melissa Bonner
Life of this patent
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Abstract
A retroviral construct harboring nucleic acids encoding a high mobility group AT-hook 2 (HMGA2) protein and lacking let-7 binding sites is described as are methods of using the retroviral vector to increase the efficacy and in vivo expansion of transduced cells in gene therapy applications.
Claims19
A retroviral construct comprising a let-7 insensitive nucleic acid encoding a high mobility group AT-hook 2 (HMGA2) protein and nucleic acids encoding, one or more therapeutic agents.
A method for increasing the efficacy and in vivo expansion of transduced cells comprising introducing a let-insensitive nucleic acid encoding a high mobility group AT-hook 2 (HMGA2) protein into a retroviral vector and transducing cells with the retroviral vector encoding the HMGA2 protein to increase the efficacy and in vivo expansion of the cells.
A method for treating a disease or condition comprising transducing cells with a retroviral vector having a let-7 insensitive nucleic acid encoding a high mobility group AT-hook 2 (HMGA2) protein and nucleic acids encoding one or more therapeutic agents and introducing the transduced cells into a subject in need of treatment with the one or more therapeutic agents thereby treating the subject's disease or condition.
A genetically-modified hematopoietic stem cell harboring a genetic alteration incurred by genome editing and including a construct having a let-7 insensitive nucleic acid encoding a high mobility group AT-hook 2 (HMGA2) protein.
A method for enhancing genome editing efficiency comprising introducing into a hematopoietic stem cell harboring a genome editing construct, a construct containing a let-7 insensitive nucleic acid encoding a HMGA2 protein thereby promoting expansion of the hematopoietic stem cell and enhancing genome editing efficiency.
A method for treating a disease or condition comprising transducing hematopoietic stem cell with a genome editing construct and a construct comprising a let-7 insensitive nucleic acid encoding a high mobility group AT-hook 2 (HMGA2) protein;
- and introducing the transduced hematopoietic stem cells into a subject in need of treatment thereby treating the subject's disease or condition.
Dependencies are inferred from the claim text.