CRISPR technology: A decade of genome editing is only the beginning

JY Wang, JA Doudna - Science, 2023 - science.org
The advent of clustered regularly interspaced short palindromic repeat (CRISPR) genome
editing, coupled with advances in computing and imaging capabilities, has initiated a new …

Drug delivery systems for RNA therapeutics

K Paunovska, D Loughrey, JE Dahlman - Nature Reviews Genetics, 2022 - nature.com
RNA-based gene therapy requires therapeutic RNA to function inside target cells without
eliciting unwanted immune responses. RNA can be ferried into cells using non-viral drug …

Current applications and future perspective of CRISPR/Cas9 gene editing in cancer

SW Wang, C Gao, YM Zheng, L Yi, JC Lu, XY Huang… - Molecular cancer, 2022 - Springer
Clustered regularly interspaced short palindromic repeats (CRISPR) system provides
adaptive immunity against plasmids and phages in prokaryotes. This system inspires the …

Drug delivery systems for CRISPR-based genome editors

V Madigan, F Zhang, JE Dahlman - Nature Reviews Drug Discovery, 2023 - nature.com
CRISPR-based drugs can theoretically manipulate any genetic target. In practice, however,
these drugs must enter the desired cell without eliciting an unwanted immune response, so …

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 …

Advances in oligonucleotide drug delivery

TC Roberts, R Langer, MJA Wood - Nature reviews Drug discovery, 2020 - nature.com
Oligonucleotides can be used to modulate gene expression via a range of processes
including RNAi, target degradation by RNase H-mediated cleavage, splicing modulation …

Engineered biomaterials for in situ tissue regeneration

AK Gaharwar, I Singh, A Khademhosseini - Nature Reviews Materials, 2020 - nature.com
In situ tissue regeneration harnesses the body's regenerative potential to control cell
functions for tissue repair. The design of biomaterials for in situ tissue engineering requires …

The promise and challenge of therapeutic genome editing

JA Doudna - Nature, 2020 - nature.com
Genome editing, which involves the precise manipulation of cellular DNA sequences to alter
cell fates and organism traits, has the potential to both improve our understanding of human …

Low immunogenicity of LNP allows repeated administrations of CRISPR-Cas9 mRNA into skeletal muscle in mice

E Kenjo, H Hozumi, Y Makita, KA Iwabuchi… - Nature …, 2021 - nature.com
Genome editing therapy for Duchenne muscular dystrophy (DMD) holds great promise,
however, one major obstacle is delivery of the CRISPR-Cas9/sgRNA system to skeletal …

Applications of genome editing technology in the targeted therapy of human diseases: mechanisms, advances and prospects

H Li, Y Yang, W Hong, M Huang, M Wu… - Signal transduction and …, 2020 - nature.com
Based on engineered or bacterial nucleases, the development of genome editing
technologies has opened up the possibility of directly targeting and modifying genomic …