Genetic engineering meets hematopoietic stem cell biology for next-generation gene therapy

S Ferrari, E Valeri, A Conti, S Scala, A Aprile… - Cell Stem Cell, 2023 - cell.com
The growing clinical success of hematopoietic stem/progenitor cell (HSPC) gene therapy
(GT) relies on the development of viral vectors as portable" Trojan horses" for safe and …

[HTML][HTML] Improving cell and gene therapy safety and performance using next-generation Nanoplasmid vectors

JA Williams, PA Paez - Molecular Therapy-Nucleic Acids, 2023 - cell.com
The cell and gene therapy industry has employed the same plasmid technology for decades
in vaccination, cell and gene therapy, and as a raw material in viral vector and RNA …

[HTML][HTML] Anti-CD117 CAR T cells incorporating a safety switch eradicate human acute myeloid leukemia and hematopoietic stem cells

CF Magnani, R Myburgh, S Brunn… - Molecular Therapy …, 2023 - cell.com
Discrimination between hematopoietic stem cells and leukemic stem cells remains a major
challenge for acute myeloid leukemia immunotherapy. CAR T cells specific for the CD117 …

Heterologous survey of 130 DNA transposons in human cells highlights their functional divergence and expands the genome engineering toolbox

T Zhang, S Tan, N Tang, Y Li, C Zhang, J Sun, Y Guo… - Cell, 2024 - cell.com
Experimental studies on DNA transposable elements (TEs) have been limited in scale,
leading to a lack of understanding of the factors influencing transposition activity …

[HTML][HTML] Ligand-based, piggyBac-engineered CAR-T cells targeting EGFR are safe and effective against non-small cell lung cancers

T Chinsuwan, K Hirabayashi, S Mishima… - Molecular Therapy …, 2023 - cell.com
Epidermal growth factor receptor (EGFR) is overexpressed in various cancers, including non-
small cell lung cancer (NSCLC), and in some somatic cells at a limited level, rendering it an …

[HTML][HTML] Chimeric antigen receptor T-cells: Emerging uses and future directions

D Dive, P Content - pharmaphorum.com
Genetic engineering technologies allow for the creation of custom-made cells that can have
transformative effects on human health. This is best exemplified by chimeric antigen receptor …