Non-Viral Delivery To Enable Genome Editing.

Drug Delivery Systems / methods Gene Editing / methods Genetic Therapy / methods Genome, Human Humans
CRISPR/Cas TALEN ZFN gene editing non-viral

Journal

Trends in biotechnology
ISSN: 1879-3096
Titre abrégé: Trends Biotechnol
Pays: England
ID NLM: 8310903

Informations de publication

Date de publication:
03 2019
Historique:
received: 06 06 2018
revised: 27 08 2018
accepted: 30 08 2018
pubmed: 4 10 2018
medline: 18 12 2019
entrez: 4 10 2018
Statut: ppublish

Résumé

Genome-editing technologies such as zinc-finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENS), and the clustered regularly interspaced short palindromic repeat (CRISPR)-associated protein system have revolutionized biological research. Each biotechnology consists of a DNA-binding protein that can be programmed to recognize and initiate double-strand breaks (DSBs) for site-specific gene modification. These technologies have the potential to be harnessed to cure diseases caused by aberrant gene expression. To be successful therapeutically, their functionality depends on their safe and efficient delivery into the cell nucleus. This review discusses the challenges in the delivery of genome-editing tools, and highlights recent innovations in non-viral delivery that have potential to overcome these limitations and advance the translation of genome editing towards patient care.

Identifiants

DOI: 10.1016/j.tibtech.2018.08.010 PMID: 30278987 PMC: PMC6378131
pubmed: 30278987
pii: S0167-7799(18)30250-6
doi: 10.1016/j.tibtech.2018.08.010
pmc: PMC6378131
mid: NIHMS1505850
pii:
doi:

Types de publication

Journal Article Research Support, N.I.H., Extramural Research Support, Non-U.S. Gov't Research Support, U.S. Gov't, Non-P.H.S. Review

Langues

eng

Sous-ensembles de citation

IM

Pagination

281-293

Subventions

Organisme : NCI NIH HHS
ID : R01 CA228133
Pays : United States
Organisme : NIBIB NIH HHS
ID : R01 EB022148
Pays : United States

Informations de copyright

Copyright © 2018 Elsevier Ltd. All rights reserved.

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Auteurs

Yuan Rui (Y)

Department of Biomedical Engineering, Johns Hopkins University School of Medicine, Baltimore, MD 21231, USA; Translational Tissue Engineering Center, Johns Hopkins University School of Medicine, Baltimore, MD 21231, USA; These authors contributed equally.

David R Wilson (DR)

Department of Biomedical Engineering, Johns Hopkins University School of Medicine, Baltimore, MD 21231, USA; Translational Tissue Engineering Center, Johns Hopkins University School of Medicine, Baltimore, MD 21231, USA; Institute for Nanobiotechnology, Johns Hopkins University School of Medicine, Baltimore, MD 21231, USA; These authors contributed equally.

Jordan J Green (JJ)

Department of Biomedical Engineering, Johns Hopkins University School of Medicine, Baltimore, MD 21231, USA; Translational Tissue Engineering Center, Johns Hopkins University School of Medicine, Baltimore, MD 21231, USA; Institute for Nanobiotechnology, Johns Hopkins University School of Medicine, Baltimore, MD 21231, USA; Departments of Materials Science and Engineering and Chemical and Biomolecular Engineering, Johns Hopkins University, Baltimore, MD 21231, USA; Departments of Ophthalmology, Oncology, and Neurosurgery, Johns Hopkins University School of Medicine, Baltimore, MD 21231, USA; Bloomberg-Kimmel Institute for Cancer Immunotherapy, Johns Hopkins University School of Medicine, Baltimore, MD 21231, USA. Electronic address: green@jhu.edu.

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Classifications MeSH

questionsmedicales.fr › Thérapeutique › Traitement médicamenteux › Systèmes de délivrance de médicaments › Non-Viral Delivery To Enable Genome Editing.
questionsmedicales.fr › Techniques d'investigation › Techniques génétiques › Génie génétique › Édition de gène › Non-Viral Delivery To Enable Genome Editing.
questionsmedicales.fr › Techniques d'investigation › Techniques génétiques › Génie génétique › Thérapie génétique › Non-Viral Delivery To Enable Genome Editing.
questionsmedicales.fr › Phénomènes génétiques › Structures génétiques › Génome › Génome humain › Non-Viral Delivery To Enable Genome Editing.
questionsmedicales.fr › Eucaryotes › Animaux › Chordés › Vertébrés › Mammifères › Eutheria › Primates › Haplorhini › Catarrhini › Hominidae › Humains › Non-Viral Delivery To Enable Genome Editing.