Precise genome-editing in human diseases: mechanisms, strategies and applications DOI Creative Commons

Yanjiang Zheng,

Yifei Li, Kaiyu Zhou

et al.

Signal Transduction and Targeted Therapy, Journal Year: 2024, Volume and Issue: 9(1)

Published: Feb. 26, 2024

Precise genome-editing platforms are versatile tools for generating specific, site-directed DNA insertions, deletions, and substitutions. The continuous enhancement of these has led to a revolution in the life sciences, which promises deliver novel therapies genetic disease. can be traced back 1950s with discovery DNA's double-helix and, after 70 years development, evolved from crude vitro applications wide range sophisticated capabilities, including vivo applications. Nonetheless, precise faces constraints such as modest efficiency, delivery challenges, off-target effects. In this review, we explore genome-editing, focus on introduction landmark events its history, various platforms, systems, First, discuss history genome-editing. Second, describe current state strategies explain how techniques offer unprecedented precision versatility modifying human genome. Third, introduce systems used deploy components through DNA, RNA, RNPs. Finally, summarize labeling endogenous genes, screening variants, molecular recording, disease models, gene therapy, ex therapy potential future advances.

Language: Английский

CRISPR technology: A decade of genome editing is only the beginning DOI
Joy Y. Wang, Jennifer A. Doudna

Science, Journal Year: 2023, Volume and Issue: 379(6629)

Published: Jan. 19, 2023

The advent of clustered regularly interspaced short palindromic repeat (CRISPR) genome editing, coupled with advances in computing and imaging capabilities, has initiated a new era which genetic diseases individual disease susceptibilities are both predictable actionable. Likewise, genes responsible for plant traits can be identified altered quickly, transforming the pace agricultural research breeding. In this Review, we discuss current state CRISPR-mediated manipulation human cells, animals, plants along relevant successes challenges present roadmap future technology.

Language: Английский

Citations

617

High-content CRISPR screening DOI Open Access
Christoph Bock, Paul Datlinger, Florence M. Chardon

et al.

Nature Reviews Methods Primers, Journal Year: 2022, Volume and Issue: 2(1)

Published: Feb. 10, 2022

Language: Английский

Citations

375

Base editing: advances and therapeutic opportunities DOI
Elizabeth M. Porto, Alexis C. Komor, Ian M. Slaymaker

et al.

Nature Reviews Drug Discovery, Journal Year: 2020, Volume and Issue: 19(12), P. 839 - 859

Published: Oct. 19, 2020

Language: Английский

Citations

351

Prime editing for precise and highly versatile genome manipulation DOI
Peter J. Chen, David R. Liu

Nature Reviews Genetics, Journal Year: 2022, Volume and Issue: 24(3), P. 161 - 177

Published: Nov. 7, 2022

Language: Английский

Citations

324

CRISPR in cancer biology and therapy DOI Open Access
Alyna Katti, Bianca J. Diaz, Christina M. Caragine

et al.

Nature reviews. Cancer, Journal Year: 2022, Volume and Issue: 22(5), P. 259 - 279

Published: Feb. 22, 2022

Language: Английский

Citations

296

Genome-wide CRISPRi/a screens in human neurons link lysosomal failure to ferroptosis DOI
Ruilin Tian,

Anthony Abarientos,

Jason Hong

et al.

Nature Neuroscience, Journal Year: 2021, Volume and Issue: 24(7), P. 1020 - 1034

Published: May 24, 2021

Language: Английский

Citations

276

The CRISPR-Cas toolbox and gene editing technologies DOI Creative Commons

Guanwen Liu,

Qiupeng Lin, Shuai Jin

et al.

Molecular Cell, Journal Year: 2021, Volume and Issue: 82(2), P. 333 - 347

Published: Dec. 29, 2021

Language: Английский

Citations

263

High-content CRISPR screening DOI
Christoph Bock, Paul Datlinger, Florence M. Chardon

et al.

Nature Reviews Methods Primers, Journal Year: 2022, Volume and Issue: 2(1)

Published: Feb. 10, 2022

Language: Английский

Citations

207

Functional interrogation of DNA damage response variants with base editing screens DOI
Raquel Cuella-Martin, Samuel B. Hayward, Xiao Fan

et al.

Cell, Journal Year: 2021, Volume and Issue: 184(4), P. 1081 - 1097.e19

Published: Feb. 1, 2021

Language: Английский

Citations

206

A new era in functional genomics screens DOI
Laralynne Przybyla, Luke A. Gilbert

Nature Reviews Genetics, Journal Year: 2021, Volume and Issue: 23(2), P. 89 - 103

Published: Sept. 20, 2021

Language: Английский

Citations

183