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 …

CRISPR technologies for precise epigenome editing

M Nakamura, Y Gao, AA Dominguez, LS Qi - Nature Cell Biology, 2021 - nature.com
The epigenome involves a complex set of cellular processes governing genomic activity.
Dissecting this complexity necessitates the development of tools capable of specifically …

The next generation of CRISPR–Cas technologies and applications

A Pickar-Oliver, CA Gersbach - Nature reviews Molecular cell biology, 2019 - nature.com
The prokaryote-derived CRISPR–Cas genome editing systems have transformed our ability
to manipulate, detect, image and annotate specific DNA and RNA sequences in living cells …

Therapeutic approaches for cardiac regeneration and repair

H Hashimoto, EN Olson, R Bassel-Duby - Nature Reviews Cardiology, 2018 - nature.com
Ischaemic heart disease is a leading cause of death worldwide. Injury to the heart is
followed by loss of the damaged cardiomyocytes, which are replaced with fibrotic scar …

Epigenome editing technologies for discovery and medicine

SR McCutcheon, D Rohm, N Iglesias… - Nature …, 2024 - nature.com
Epigenome editing has rapidly evolved in recent years, with diverse applications that
include elucidating gene regulation mechanisms, annotating coding and noncoding …

In vivo simultaneous transcriptional activation of multiple genes in the brain using CRISPR–dCas9-activator transgenic mice

H Zhou, J Liu, C Zhou, N Gao, Z Rao, H Li, X Hu… - Nature …, 2018 - nature.com
Despite rapid progresses in the genome-editing field, in vivo simultaneous overexpression
of multiple genes remains challenging. We generated a transgenic mouse using an …

[HTML][HTML] CRISPR/Cas9-based engineering of the epigenome

J Pulecio, N Verma, E Mejía-Ramírez, D Huangfu… - Cell stem cell, 2017 - cell.com
Determining causal relationships between distinct chromatin features and gene expression,
and ultimately cell behavior, remains a major challenge. Recent developments in targetable …

Multiplexed genome regulation in vivo with hyper-efficient Cas12a

LY Guo, J Bian, AE Davis, P Liu, HR Kempton… - Nature cell …, 2022 - nature.com
Multiplexed modulation of endogenous genes is crucial for sophisticated gene therapy and
cell engineering. CRISPR–Cas12a systems enable versatile multiple-genomic-loci targeting …

Refactoring transcription factors for metabolic engineering

C Deng, Y Wu, X Lv, J Li, Y Liu, G Du, J Chen… - Biotechnology …, 2022 - Elsevier
Due to the ability to regulate target metabolic pathways globally and dynamically, metabolic
regulation systems composed of transcription factors have been widely used in metabolic …

Transgenic mice for in vivo epigenome editing with CRISPR-based systems

MP Gemberling, K Siklenka, E Rodriguez… - Nature …, 2021 - nature.com
CRISPR-Cas9 technologies have dramatically increased the ease of targeting DNA
sequences in the genomes of living systems. The fusion of chromatin-modifying domains to …