R. Scott McIvor

The general research interest of Dr. McIvor's laboratory is genetic therapy for inherited diseases and cancer. Specific research activities include: (i) Use of recombinant lentiviral vectors for gene transfer into hematopoietic stem cells in the treatment of cancer and inherited disorders such as immunodeficiency diseases and lysosomal storage diseases; (ii) Use of adeno-associated virus for treatment of storage diseases and as a vector for gene transfer into the central nervous system in the treatment of neurological disorders; (iii) Adaptation of an active vertebrate transposon ("Sleeping Beauty") in the development non-viral genetic therapies; (ii) Chromosomal site directed correction of mutations in hematopoietic stem cells and other target tissues for the treatment of immunodeficiencies and other inherited disorders. Recent highlights have included restoration of immunity in infants with severe combined immunodeficiency (SCID), preclinical work supporting the first human clinical trial of in vivo gene editing, and preclinical work supporting clinical trials of new genetic therapies for the mucopolysaccharidoses MPS I and MPS II.