Alexion Pharmaceuticals, Inc.

Scott P Kennedy

James M Mason

Seth A Fidel

Retroviral vector particles are provided which contain: 1) oncoretroviral gag, pol, and env proteins, including an oncoretroviral gag capsid protein which has been mutated so as to contain a nuclear localization signal (NLS) sequence; and 2) at least one exogenous gene. The particles can be used to transduce non-proliferating cells, including stem cells and neurons. The presence of the NLS sequence allows the at least one exogenous gene to enter into the nucleus of a target cell, thus allowing integration of the gene into the genome of the target cell.

Retroviral vector particles for transducing non-proliferating cells

Retroviral vector particles are provided which contain: 1) oncoretroviral gag, pol, and env proteins, including an oncoretroviral gag matrix protein which has been mutated so as to contain a nuclear localization signal (NLS) sequence; and 2) at least one exogenous gene. The particles can be used to transduce non-proliferating cells, including stem cells and neurons. The presence of the NLS sequence allows the at least one exogenous gene to enter into the nucleus of a target cell, thus allowing integration of the gene into the genome of the target cell.

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Southington, CT, United States of America

Scott P Kennedy