Intracellular delivery by membrane disruption: mechanisms, strategies, and concepts

MP Stewart, R Langer, KF Jensen - Chemical reviews, 2018 - ACS Publications
Intracellular delivery is a key step in biological research and has enabled decades of
biomedical discoveries. It is also becoming increasingly important in industrial and medical …

The expanding role for small molecules in immuno-oncology

R Offringa, L Kötzner, B Huck, K Urbahns - Nature Reviews Drug …, 2022 - nature.com
The advent of immune checkpoint inhibition (ICI) using antibodies against PD1 and its
ligand PDL1 has prompted substantial efforts to develop complementary drugs. Although …

[HTML][HTML] Non-viral, specifically targeted CAR-T cells achieve high safety and efficacy in B-NHL

J Zhang, Y Hu, J Yang, W Li, M Zhang, Q Wang… - Nature, 2022 - nature.com
Recently, chimeric antigen receptor (CAR)-T cell therapy has shown great promise in
treating haematological malignancies,,,,,–. However, CAR-T cell therapy currently has …

High-efficiency transgene integration by homology-directed repair in human primary cells using DNA-PKcs inhibition

S Selvaraj, WN Feist, S Viel, S Vaidyanathan… - Nature …, 2024 - nature.com
Therapeutic applications of nuclease-based genome editing would benefit from improved
methods for transgene integration via homology-directed repair (HDR). To improve HDR …

A high-fidelity Cas9 mutant delivered as a ribonucleoprotein complex enables efficient gene editing in human hematopoietic stem and progenitor cells

CA Vakulskas, DP Dever, GR Rettig, R Turk… - Nature medicine, 2018 - nature.com
Translation of the CRISPR–Cas9 system to human therapeutics holds high promise.
However, specificity remains a concern especially when modifying stem cell populations …

Targeting a CAR to the TRAC locus with CRISPR/Cas9 enhances tumour rejection

J Eyquem, J Mansilla-Soto, T Giavridis… - Nature, 2017 - nature.com
Chimeric antigen receptors (CARs) are synthetic receptors that redirect and reprogram T
cells to mediate tumour rejection. The most successful CARs used to date are those …

Reprogramming human T cell function and specificity with non-viral genome targeting

TL Roth, C Puig-Saus, R Yu, E Shifrut, J Carnevale… - Nature, 2018 - nature.com
Decades of work have aimed to genetically reprogram T cells for therapeutic purposes,
using recombinant viral vectors, which do not target transgenes to specific genomic sites …

Programming CAR T cell tumor recognition: tuned antigen sensing and logic gating

M Hamieh, J Mansilla-Soto, I Rivière, M Sadelain - Cancer Discovery, 2023 - AACR
The success of chimeric antigen receptor (CAR) T cells targeting B-cell malignancies
propelled the field of synthetic immunology and raised hopes to treat solid tumors in a …

In vitro and ex vivo strategies for intracellular delivery

MP Stewart, A Sharei, X Ding, G Sahay, R Langer… - Nature, 2016 - nature.com
Intracellular delivery of materials has become a critical component of genome-editing
approaches, ex vivo cell-based therapies, and a diversity of fundamental research …

Intratumoral activation of the necroptotic pathway components RIPK1 and RIPK3 potentiates antitumor immunity

AG Snyder, NW Hubbard, MN Messmer… - Science …, 2019 - science.org
Although the signaling events that induce different forms of programmed cell death are well
defined, the subsequent immune responses to dying cells in the context of cancer remain …