Prime editing for precise and highly versatile genome manipulation

PJ Chen, DR Liu - Nature Reviews Genetics, 2023 - nature.com
Programmable gene-editing tools have transformed the life sciences and have shown
potential for the treatment of genetic disease. Among the CRISPR–Cas technologies that …

RNA-based therapeutics: an overview and prospectus

Y Zhu, L Zhu, X Wang, H Jin - Cell death & disease, 2022 - nature.com
The growing understanding of RNA functions and their crucial roles in diseases promotes
the application of various RNAs to selectively function on hitherto “undruggable” proteins …

mRNA-based cancer therapeutics

C Liu, Q Shi, X Huang, S Koo, N Kong, W Tao - Nature Reviews Cancer, 2023 - nature.com
Due to the fact that mRNA technology allows the production of diverse vaccines and
treatments in a shorter time frame and with reduced expense compared to conventional …

CRISPR/Cas9 therapeutics: progress and prospects

T Li, Y Yang, H Qi, W Cui, L Zhang, X Fu, X He… - … and Targeted Therapy, 2023 - nature.com
Clustered regularly interspaced short palindromic repeats (CRISPR)/CRISPR-associated
protein 9 (Cas9) gene-editing technology is the ideal tool of the future for treating diseases …

CRISPR in cancer biology and therapy

A Katti, BJ Diaz, CM Caragine, NE Sanjana… - Nature Reviews …, 2022 - nature.com
Over the past decade, CRISPR has become as much a verb as it is an acronym,
transforming biomedical research and providing entirely new approaches for dissecting all …

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 …

Roles of transposable elements in the regulation of mammalian transcription

R Fueyo, J Judd, C Feschotte, J Wysocka - Nature reviews Molecular …, 2022 - nature.com
Transposable elements (TEs) comprise about half of the mammalian genome. TEs often
contain sequences capable of recruiting the host transcription machinery, which they use to …

[HTML][HTML] The CRISPR-Cas toolbox and gene editing technologies

G Liu, Q Lin, S Jin, C Gao - Molecular cell, 2022 - cell.com
The emergence of CRISPR-Cas systems has accelerated the development of gene editing
technologies, which are widely used in the life sciences. To improve the performance of …

Assessing and advancing the safety of CRISPR-Cas tools: from DNA to RNA editing

J Tao, DE Bauer, R Chiarle - Nature Communications, 2023 - nature.com
CRISPR-Cas gene editing has revolutionized experimental molecular biology over the past
decade and holds great promise for the treatment of human genetic diseases. Here we …

Human organoids: model systems for human biology and medicine

J Kim, BK Koo, JA Knoblich - Nature Reviews Molecular Cell Biology, 2020 - nature.com
The historical reliance of biological research on the use of animal models has sometimes
made it challenging to address questions that are specific to the understanding of human …