IMPROVED METHODS AND MEANS FOR LENTIVIRAL GENE DELIVERY

摘要

The invention relates to the field of gene therapy and more in specific to lentiviral gene delivery vehicles and methods for efficient transduction of lentiviral gene delivery vehicles into hematopoietic stem cells and their descendants. Preferably, the invention provides in one of its embodiments a method of gene transfer into e.g. pluripotent hematopoietic stem cells and their descendants, enabling successful transduction of cells, including transplantable cell populations comprising hematopoietic stem cells that give rise to progeny expressing the transduced gene(s). The invention further comprises a method for treating a variety of hereditary and acquired human disease by transfer of therapeutically active genes into hematopoietic stem cells. As a non-limiting example, the invention shows that symptoms associated with Pompe disease are (completely) reduced and/or alleviated by treatment of a subject suffering from Pompe disease withJiematopoietic stem cell transduced with an alpha- glucosidase comprising lentiviral vector.