Under his direction, plasmid DNA, CAR - T - cells, regulatory T - cells, engineered stem cell grafts and gene
modified hematopoietic stem cell products have been manufactured and released for use in Phase I / II clinical trials.
Several gene therapy clinical trials using gene -
modified hematopoietic stem cells have shown therapeutic efficacy in multiple disease areas.
However, coreceptor - specific ZFNs represent a novel therapeutic approach to recapitulate this success via autologous transplantation of gene -
modified hematopoietic stem cells and mature CD4 + T cells.
Not exact matches
Beilhack and colleagues found that a slightly
modified version of STAR2 has a similar effect on human T reg cells, suggesting that the approach could also prevent GvHD in leukemia and lymphoma patients after bone marrow or
hematopoietic stem cell transplants.
Another
modified peptide POL6326 is in phase II clinical trial in
hematopoietic stem cell mobilization in multiple myeloma [128].
Successful treatment of murine ß - thalassemia using in vivo selection of genetically -
modified, drug resistant
hematopoietic cells.
In order to
modify or expand primary human
hematopoietic cells, they must first be isolated from the body and then cultured under conditions that allow for their modification.
Lentiviral - based gene therapy methods to
modify human CD34 +
hematopoietic stem cells have been investigated as a way of treating various hematological disorders including X-linked chronic granulomatous disease (X-CGD).