Safe harbours for the integration of new DNA in the human genome

M Sadelain, EP Papapetrou, FD Bushman - Nature Reviews Cancer, 2012 - nature.com
Interactions between newly integrate DNA and the host genome limit the reliability and
safety of transgene integration for therapeutic cell engineering and other applications …

Gene insertion into genomic safe harbors for human gene therapy

EP Papapetrou, A Schambach - Molecular Therapy, 2016 - cell.com
Genomic safe harbors (GSHs) are sites in the genome able to accommodate the integration
of new genetic material in a manner that ensures that the newly inserted genetic elements:(i) …

Rapid protein depletion in human cells by auxin-inducible degron tagging with short homology donors

T Natsume, T Kiyomitsu, Y Saga, MT Kanemaki - Cell reports, 2016 - cell.com
Studying the role of essential proteins is dependent upon a method for rapid inactivation, in
order to study the immediate phenotypic consequences. Auxin-inducible degron (AID) …

Nonviral gene editing via CRISPR/Cas9 delivery by membrane-disruptive and endosomolytic helical polypeptide

HX Wang, Z Song, YH Lao, X Xu… - Proceedings of the …, 2018 - National Acad Sciences
Effective and safe delivery of the CRISPR/Cas9 gene-editing elements remains a challenge.
Here we report the development of PEGylated nanoparticles (named P-HNPs) based on the …

An iCRISPR platform for rapid, multiplexable, and inducible genome editing in human pluripotent stem cells

F González, Z Zhu, ZD Shi, K Lelli, N Verma, QV Li… - Cell stem cell, 2014 - cell.com
Human pluripotent stem cells (hPSCs) offer a unique platform for elucidating the genes and
molecular pathways that underlie complex traits and diseases. To realize this promise …

Engineering chimeric antigen receptor neutrophils from human pluripotent stem cells for targeted cancer immunotherapy

Y Chang, R Syahirah, X Wang, G Jin… - Cell reports, 2022 - cell.com
Neutrophils, the most abundant white blood cells in circulation, are closely related to cancer
development and progression. Healthy primary neutrophils present potent cytotoxicity …

TALEN and CRISPR/Cas genome editing systems: tools of discovery

AA Nemudryi, KR Valetdinova… - Acta Naturae …, 2014 - cyberleninka.ru
Precise studies of plant, animal and human genomes enable remarkable opportunities of
obtained data application in biotechnology and medicine. However, knowing nucleotide …

Efficient targeting of expressed and silent genes in human ESCs and iPSCs using zinc-finger nucleases

D Hockemeyer, F Soldner, C Beard, Q Gao… - Nature …, 2009 - nature.com
Realizing the full potential of human embryonic stem cells (hESCs) and induced pluripotent
stem cells (hiPSCs) requires efficient methods for genetic modification. However, techniques …

Meganucleases and other tools for targeted genome engineering: perspectives and challenges for gene therapy

G Silva, L Poirot, R Galetto, J Smith… - Current gene …, 2011 - ingentaconnect.com
The importance of safer approaches for gene therapy has been underscored by a series of
severe adverse events (SAEs) observed in patients involved in clinical trials for Severe …

[HTML][HTML] High-efficiency nonviral CRISPR/Cas9-mediated gene editing of human T cells using plasmid donor DNA

SA Oh, K Senger, S Madireddi… - Journal of Experimental …, 2022 - rupress.org
High-efficiency nonviral CRISPR/Cas9-mediated gene editing of human T cells using plasmid
donor DNA | Journal of Experimental Medicine | Rockefeller University Press Skip to Main …