Fifteen years ago, the drive to study retroviruses was rooted principally in the traditional goal
of using animal models to understand human cancer. The historical importance of …

The concept of gene therapy originated in the mid twentieth century and was perceived as a
revolutionary technology with the promise to cure almost any disease of which the molecular …

Adenosine deaminase (ADA) deficiency results in severe combined immunodeficiency, the
first genetic disorder treated by gene therapy. Two different retroviral vectors were used to …

HEMATOPOIESIS IS A life-long process responsible for replenishing both hematopoietic
progenitor cells and mature blood cells from a pool of pluripotent, long-term reconstituting …

''It seems more than ever likely that blood-derived stem tive capacity. 47-49 In the allogeneic
setting, mobilized blood cells have also been used instead of BM. 50 Umbilical cord cells …

The development of stem–cell gene therapy is hindered by the absence of repopulation
assays for primitive human hematopoietic cells. Current methods of gene transfer rely on in …

Hematopoietic cells are important targets for genetic modification with retroviral vectors.
Attempts at human gene therapy of stem cells have achieved limited success partly because …

Human hematopoiesis originates in a population of stem cells with transplantable lympho-
myeloid reconstituting potential, but a method for quantitating such cells has not been …

The use of Moloney murine leukemia virus (Mo-MLV)-based vectors to deliver therapeutic
genes into target cells is limited by their inability to transduce nondividing cells. To test the …

Little is known about the potential for engraftment of autologous hematopoietic stem cells in
human adults not subjected to myeloablative conditioning regimens. Five adult patients with …