TY  - GEN
ID  - heidok33156
KW  - iPSC
KW  -  S/MAR DNA Vector
KW  -  gene therapy
KW  -  cell therapy
KW  -  T-cell
KW  -  immunotherapy
KW  -  HSPC
KW  -  HSC
KW  -  DNA Vector Lab
KW  -  iPSC-derived
AV  - public
CY  - Heidelberg
TI  - Developments Toward an iPSC-Derived T-Cell Immunotherapy Using S/MAR DNA Vectors: A Focus on Hematopoietic Stem and Progenitor Cells
Y1  - 2024///
N2  - Randomly integrating viral vectors pose a genotoxic risk when used as a genetic modification tool. Our lab has developed a non-integrating DNA vector (S/MAR DNA vector) that persists episomally in cells and provides long-term transgene expression similar to integrating viral vectors. In this project, I show a proof of concept of developing an
off-the-shelf iPSC-derived T-cell immunotherapy using S/MAR DNA vectors. I developed an S/MAR DNA vector that is optimal for T-cells as well as iPSC genetic modification. I
created a platform to screen vector features in mouse hematopoietic stem and progenitor cells. I indicate that iPSC lines that were genetically modified using S/MAR DNA vectors can differentiate into hematopoietic stem and progenitor-like cells, and I compared it with an iPSC line genetically modified using a lentiviral vector. Finally, this report shows that iPSC lines expressing a CAR can differentiate into phenotypic T-cells.

The personalization of gene and cell therapies is expensive. They may become more affordable and accessible to patients when iPSCs are used to generate allogeneic cell therapies. Exploiting iPSCs could reduce the cost, offer a healthier cell source, provide a quicker treatment option, and offer a more standardized therapy to patients. The data described in
this report suggest the possibility of generating iPSC-derived T-cell immunotherapies utilizing S/MAR DNA vectors.
A1  - Peterson, Julia
UR  - https://archiv.ub.uni-heidelberg.de/volltextserver/33156/
ER  -