mRNA-based therapeutics: powerful and versatile tools to combat diseases

S Qin, X Tang, Y Chen, K Chen, N Fan… - Signal transduction and …, 2022 - nature.com
The therapeutic use of messenger RNA (mRNA) has fueled great hope to combat a wide
range of incurable diseases. Recent rapid advances in biotechnology and molecular …

[HTML][HTML] CRISPR-Cas12a: Functional overview and applications

B Paul, G Montoya - Biomedical journal, 2020 - Elsevier
Prokaryotes have developed an adaptive immune system called Clustered regularly
interspaced short palindromic repeats (CRISPR) to combat attacks by foreign mobile genetic …

Allogeneic CAR-T cells: more than ease of access?

C Graham, A Jozwik, A Pepper, R Benjamin - Cells, 2018 - mdpi.com
Patient derived anti-CD19 chimeric antigen receptor-T (CAR-T) cells are a powerful tool in
achieving a complete remission in a range of B-cell malignancies, most notably B-acute …

Ready for repair? Gene editing enters the clinic for the treatment of human disease

MPT Ernst, M Broeders, P Herrero-Hernandez… - … Therapy Methods & …, 2020 - cell.com
We present an overview of clinical trials involving gene editing using clustered interspaced
short palindromic repeats (CRISPR)-CRISPR-associated protein 9 (Cas9), transcription …

The quest for the best: how TCR affinity, avidity, and functional avidity affect TCR-engineered T-cell antitumor responses

D Campillo-Davo, D Flumens, E Lion - Cells, 2020 - mdpi.com
Over the past decades, adoptive transfer of T cells has revolutionized cancer
immunotherapy. In particular, T-cell receptor (TCR) engineering of T cells has marked …

Long terminal repeat CRISPR-CAR-coupled “universal” T cells mediate potent anti-leukemic effects

C Georgiadis, R Preece, L Nickolay, A Etuk, A Petrova… - Molecular Therapy, 2018 - cell.com
Gene editing can be used to overcome allo-recognition, which otherwise limits allogeneic T
cell therapies. Initial proof-of-concept applications have included generation of such" …

CRISPR-mediated integration of large gene cassettes using AAV donor vectors

RO Bak, MH Porteus - Cell reports, 2017 - cell.com
The CRISPR/Cas9 system has recently been shown to facilitate high levels of precise
genome editing using adeno-associated viral (AAV) vectors to serve as donor template DNA …

Engineering strategies to enhance TCR-based adoptive T cell therapy

JA Rath, C Arber - Cells, 2020 - mdpi.com
T cell receptor (TCR)-based adoptive T cell therapies (ACT) hold great promise for the
treatment of cancer, as TCRs can cover a broad range of target antigens. Here we …

[HTML][HTML] mRNA therapeutics: New vaccination and beyond

HH Wei, L Zheng, Z Wang - Fundamental Research, 2023 - Elsevier
The idea of mRNA therapy had been conceived for decades before it came into reality
during the Covid-19 pandemic. The mRNA vaccine emerges as a powerful and general tool …

Variability in genome editing outcomes: challenges for research reproducibility and clinical safety

L Teboul, Y Herault, S Wells, W Qasim, G Pavlovic - Molecular Therapy, 2020 - cell.com
Genome editing tools have already revolutionized biomedical research and are also
expected to have an important impact in the clinic. However, their extensive use in research …