Chimeric antigen receptor natural killer (CAR-NK) cell design and engineering for cancer therapy
Due to their efficient recognition and lysis of malignant cells, natural killer (NK) cells are
considered as specialized immune cells that can be genetically modified to obtain capable …
considered as specialized immune cells that can be genetically modified to obtain capable …
Gene therapy using haematopoietic stem and progenitor cells
G Ferrari, AJ Thrasher, A Aiuti - Nature Reviews Genetics, 2021 - nature.com
Haematopoietic stem and progenitor cell (HSPC) gene therapy has emerged as an effective
treatment modality for monogenic disorders of the blood system such as primary …
treatment modality for monogenic disorders of the blood system such as primary …
High-efficiency transgene integration by homology-directed repair in human primary cells using DNA-PKcs inhibition
Therapeutic applications of nuclease-based genome editing would benefit from improved
methods for transgene integration via homology-directed repair (HDR). To improve HDR …
methods for transgene integration via homology-directed repair (HDR). To improve HDR …
Choice of template delivery mitigates the genotoxic risk and adverse impact of editing in human hematopoietic stem cells
Long-range gene editing by homology-directed repair (HDR) in hematopoietic
stem/progenitor cells (HSPCs) often relies on viral transduction with recombinant adeno …
stem/progenitor cells (HSPCs) often relies on viral transduction with recombinant adeno …
Cas9 activates the p53 pathway and selects for p53-inactivating mutations
Cas9 is commonly introduced into cell lines to enable CRISPR–Cas9-mediated genome
editing. Here, we studied the genetic and transcriptional consequences of Cas9 expression …
editing. Here, we studied the genetic and transcriptional consequences of Cas9 expression …
Precise gene editing preserves hematopoietic stem cell function following transient p53-mediated DNA damage response
Precise gene editing in hematopoietic stem and progenitor cells (HSPCs) holds promise for
treating genetic diseases. However, responses triggered by programmable nucleases in …
treating genetic diseases. However, responses triggered by programmable nucleases in …
Genetic engineering meets hematopoietic stem cell biology for next-generation gene therapy
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 …
(GT) relies on the development of viral vectors as portable" Trojan horses" for safe and …
TP53-dependent toxicity of CRISPR/Cas9 cuts is differential across genomic loci and can confound genetic screening
CRISPR/Cas9 gene editing can inactivate genes in a precise manner. This process involves
DNA double-strand breaks (DSB), which may incur a loss of cell fitness. We hypothesize that …
DNA double-strand breaks (DSB), which may incur a loss of cell fitness. We hypothesize that …
[HTML][HTML] DNA damage contributes to neurotoxic inflammation in Aicardi-Goutières syndrome astrocytes
AMS Giordano, M Luciani, F Gatto… - Journal of Experimental …, 2022 - rupress.org
Aberrant induction of type I IFN is a hallmark of the inherited encephalopathy Aicardi-
Goutières syndrome (AGS), but the mechanisms triggering disease in the human central …
Goutières syndrome (AGS), but the mechanisms triggering disease in the human central …
Gene therapy targeting haematopoietic stem cells for inherited diseases: progress and challenges
M Cavazzana, FD Bushman, A Miccio… - Nature reviews Drug …, 2019 - nature.com
Pioneering gene therapy trials have shown that the genetic engineering of haematopoietic
stem and progenitor cells can be an alternative to allogeneic transplantation in the treatment …
stem and progenitor cells can be an alternative to allogeneic transplantation in the treatment …