A highly efficient transgene knock-in technology in clinically relevant cell types
…, D Zhang, JD Nelson, P Zhang, P Mazzucato… - Nature …, 2024 - nature.com
Inefficient knock-in of transgene cargos limits the potential of cell-based medicines. In this
study, we used a CRISPR nuclease that targets a site within an exon of an essential gene and …
study, we used a CRISPR nuclease that targets a site within an exon of an essential gene and …
Multiplexed DNA repair assays for multiple lesions and multiple doses via transcription inhibition and transcriptional mutagenesis
The capacity to repair different types of DNA damage varies among individuals, making
them more or less susceptible to the detrimental health consequences of damage exposures. …
them more or less susceptible to the detrimental health consequences of damage exposures. …
Minor changes in expression of the mismatch repair protein MSH2 exert a major impact on glioblastoma response to temozolomide
Glioblastoma (GBM) is often treated with the cytotoxic drug temozolomide, but the disease
inevitably recurs in a drug-resistant form after initial treatment. Here, we report that in GBM …
inevitably recurs in a drug-resistant form after initial treatment. Here, we report that in GBM …
DNA repair capacity in multiple pathways predicts chemoresistance in glioblastoma multiforme
Cancer cells can resist the effects of DNA-damaging therapeutic agents via utilization of DNA
repair pathways, suggesting that DNA repair capacity (DRC) measurements in cancer cells …
repair pathways, suggesting that DNA repair capacity (DRC) measurements in cancer cells …
In vivo measurements of interindividual differences in DNA glycosylases and APE1 activities
…, ZD Nagel, JJ Jordan, P Mazzucato… - Proceedings of the …, 2017 - National Acad Sciences
The integrity of our DNA is challenged with at least 100,000 lesions per cell on a daily basis.
Failure to repair DNA damage efficiently can lead to cancer, immunodeficiency, and …
Failure to repair DNA damage efficiently can lead to cancer, immunodeficiency, and …
[HTML][HTML] A multiplexed gRNA piggyBac transposon system facilitates efficient induction of CRISPRi and CRISPRa in human pluripotent stem cells
DZ Hazelbaker, A Beccard, G Angelini, P Mazzucato… - Scientific reports, 2020 - nature.com
CRISPR-Cas9-mediated gene interference (CRISPRi) and activation (CRISPRa) approaches
hold promise for functional gene studies and genome-wide screens in human pluripotent …
hold promise for functional gene studies and genome-wide screens in human pluripotent …
[PDF][PDF] De novo DNA methyltransferases DNMT3A and DNMT3B are essential for XIST silencing for erosion of dosage compensation in pluripotent stem cells
Human pluripotent stem cells (hPSCs) have proven to be valuable tools for both drug discovery
and the development of cell-based therapies. However, the long non-coding RNA XIST, …
and the development of cell-based therapies. However, the long non-coding RNA XIST, …
[PDF][PDF] Molecular convergence between Down syndrome and fragile X syndrome identified using human pluripotent stem cell models
SG Susco, S Ghosh, P Mazzucato, G Angelini… - Cell reports, 2022 - cell.com
Down syndrome (DS), driven by an extra copy of chromosome 21 (HSA21), and fragile X
syndrome (FXS), driven by loss of the RNA-binding protein FMRP, are two common genetic …
syndrome (FXS), driven by loss of the RNA-binding protein FMRP, are two common genetic …
CometChip analysis of human primary lymphocytes enables quantification of inter-individual differences in the kinetics of repair of oxidative DNA damage
LP Ngo, S Kaushal, IA Chaim, P Mazzucato… - Free Radical Biology …, 2021 - Elsevier
Although DNA repair is known to impact susceptibility to cancer and other diseases, relatively
few population studies have been performed to evaluate DNA repair kinetics in people …
few population studies have been performed to evaluate DNA repair kinetics in people …
[PDF][PDF] A scaled framework for CRISPR editing of human pluripotent stem cells to study psychiatric disease
…, AM Bara, N Dabkowski, A Messana, P Mazzucato… - Stem cell reports, 2017 - cell.com
Scaling of CRISPR-Cas9 technology in human pluripotent stem cells (hPSCs) represents an
important step for modeling complex disease and developing drug screens in human cells. …
important step for modeling complex disease and developing drug screens in human cells. …