Engineering the next generation of cell-based therapeutics

CJ Bashor, IB Hilton, H Bandukwala… - Nature Reviews Drug …, 2022 - nature.com
Cell-based therapeutics are an emerging modality with the potential to treat many currently
intractable diseases through uniquely powerful modes of action. Despite notable recent …

Engineering living therapeutics with synthetic biology

A Cubillos-Ruiz, T Guo, A Sokolovska… - Nature Reviews Drug …, 2021 - nature.com
The steadfast advance of the synthetic biology field has enabled scientists to use genetically
engineered cells, instead of small molecules or biologics, as the basis for the development …

Immuno-oncology: emerging targets and combination therapies

HT Marshall, MBA Djamgoz - Frontiers in oncology, 2018 - frontiersin.org
Host immunity recognizes and eliminates most early tumor cells, yet immunological
checkpoints, exemplified by CTLA-4, PD-1, and PD-L1, pose a significant obstacle to …

Systems and synthetic microRNA biology: from biogenesis to disease pathogenesis

H Matsuyama, HI Suzuki - International journal of molecular sciences, 2019 - mdpi.com
MicroRNAs (miRNAs) are approximately 22-nucleotide-long, small non-coding RNAs that
post-transcriptionally regulate gene expression. The biogenesis of miRNAs involves multiple …

Designing cell function: assembly of synthetic gene circuits for cell biology applications

M **e, M Fussenegger - Nature Reviews Molecular Cell Biology, 2018 - nature.com
Synthetic biology is the discipline of engineering application-driven biological functionalities
that were not evolved by nature. Early breakthroughs of cell engineering, which were based …

Synthetic introns enable splicing factor mutation-dependent targeting of cancer cells

K North, S Benbarche, B Liu, J Pangallo, S Chen… - Nature …, 2022 - nature.com
Many cancers carry recurrent, change-of-function mutations affecting RNA splicing factors.
Here, we describe a method to harness this abnormal splicing activity to drive splicing factor …

A pan-cancer transcriptome analysis reveals pervasive regulation through alternative promoters

D Demircioğlu, E Cukuroglu, M Kindermans, T Nandi… - Cell, 2019 - cell.com
Most human protein-coding genes are regulated by multiple, distinct promoters, suggesting
that the choice of promoter is as important as its level of transcriptional activity. However …

Engineered dCas9 with reduced toxicity in bacteria: implications for genetic circuit design

S Zhang, CA Voigt - Nucleic acids research, 2018 - academic.oup.com
Large synthetic genetic circuits require the simultaneous expression of many regulators.
Deactivated Cas9 (dCas9) can serve as a repressor by having a small guide RNA (sgRNA) …

Nanotechnology‐Assisted RNA Delivery: From Nucleic Acid Therapeutics to COVID‐19 Vaccines

C Rinoldi, SS Zargarian, P Nakielski, X Li… - Small …, 2021 - Wiley Online Library
In recent years, the main quest of science has been the pioneering of the groundbreaking
biomedical strategies needed for achieving a personalized medicine. Ribonucleic acids …

N 1-Methylpseudouridine substitution enhances the performance of synthetic mRNA switches in cells

CJC Parr, S Wada, K Kotake, S Kameda… - Nucleic Acids …, 2020 - academic.oup.com
Synthetic messenger RNA (mRNA) tools often use pseudouridine and 5-methyl cytidine as
substitutions for uridine and cytidine to avoid the immune response and cytotoxicity induced …