Discover Community Create You

Loading publication...

Noodle always counts identifier-free feature events for service operation. With your permission, Google Analytics can also estimate audience and engagement. Search text, publication identifiers, cookies, IP addresses and browser fingerprints are never included in Noodle's first-party event counters.

Privacy details

Article

Modulating gene regulation to treat genetic disorders.

Nature reviews. Drug discovery - 1 Nov 2020

Matharu Navneet, Ahituv Nadav

Abstract excerpt

Over a thousand diseases are caused by mutations that alter gene expression levels. The potential of nuclease-deficient zinc fingers, TALEs or CRISPR fusion systems to treat these diseases by modulating gene expression has recently emerged. These systems can be applied to modify the activity of gene-regulatory elements - promoters, enhancers, silencers and insulators, subsequently changing their target gene...

Read the complete abstract on PubMed

Topics

Share this publication in a Topic to start or enrich a Post.

Share to CRISPR-Cas Systems
Share to Gene Expression Regulation
Share to Genetic Diseases, Inborn

Methodology Data sources Editorial policy Privacy About Contact Helena Bioinformatics

Share to Mutation

Share to Pharmaceutical Preparations

Source

Open in PubMed Open full text

Identifiers

PMID: 33020616
DOI: 10.1038/s41573-020-0083-7
PMCID: PMC12922764

Provenance

PubMed metadata and Literature Corpus relationships. Corpus snapshot updated 6 Sept 2026. Manifest publication-graph-cohort-2026-09-06.1.

Source receipt: pubmed, retrieved 6 Sept 2026.

Related research

Semantic proximity does not establish scientific evidence.

Research trail→

Click a neighbor to travelStep 1 · 12 closest

Modulating gene regulation to treat genetic disorders.PMID 33020616Read ↗

CRISPR system-mediated gene therapy: how flexible it used in genetic diseasesSemantic proximity Read ↗

Next stop for the CRISPR revolution: RNA-guided epigenetic regulators.Semantic proximity Read ↗

Gene and cell therapy of human genetic diseases: Recent advances and future directionsSemantic proximity Read ↗

Genome-editing Technologies for Gene and Cell TherapySemantic proximity Read ↗

Using CRISPR to understand and manipulate gene regulation.Semantic proximity Read ↗

Zinc Finger Nucleases as tools to understand and treat human diseasesSemantic proximity Read ↗

Gene, Stem Cell, and Future Therapies for Orphan DiseasesSemantic proximity Read ↗

Natural and Experimental Rewiring of Gene Regulatory Regions.Semantic proximity Read ↗

Epigenome editing for targeted DNA (de)methylation: a new perspective in modulating gene expressionSemantic proximity Read ↗

CRISPR base editing of cis-regulatory elements enables the perturbation of neurodegeneration-linked genesSemantic proximity Read ↗

Gene editing and gene regulation with CRISPR.Semantic proximity Read ↗

Inducible, tunable and multiplex human gene regulation using CRISPR-Cpf1-based transcription factorsSemantic proximity Read ↗

⌁Select a neighboring publication to make it the new centre.