Cord blood transplantation and the potential for gene therapy. Gene transduction using a recombinant adeno-associated viral vector.
Cord blood, which contains a high frequency of immature stem/progenitor cells with extensive proliferative and replating capacity in vitro was used as a clinical source of transplantable stem and progenitor cells. These cells can be efficiently transduced with new genetic material by using AAV or retroviral vectors. Using a recombinant AAV vector, high level expression of the lacZ gene under a CMV promoter was demonstrated in immature subsets of cord blood progenitor cells.
Broxmeyer
H E
HE
Department of Medicine (Hematology/Oncology), Walther Oncology Center, Indianapolis, Indiana 46202-5121, USA.
Cooper
S
S
Etienne-Julan
M
M
Wang
X S
XS
Ponnazhagan
S
S
Braun
S
S
Lu
L
L
Srivastava
A
A
D004274:DNA, Recombinant; D000229:Dependovirus / Q000235:genetics*; D005312:Fetal Blood / Q000166:cytology*; D015870:Gene Expression; D018014:Gene Transfer Techniques; D015316:Genetic Therapy; D005822:Genetic Vectors; D006098:Granulocytes / Q000378:metabolism; D018380:Hematopoietic Stem Cell Transplantation; D006412:Hematopoietic Stem Cells / Q000378:metabolism; D006801:Humans; D007231:Infant, Newborn; D001616:beta-Galactosidase / Q000235:genetics
D004274:DNA, Recombinant; D001616:beta-Galactosidase