Adenoviral vectors for in vivo delivery of CRISPR-Cas gene editors.

CRISPR-Cas Systems Clustered Regularly Interspaced Short Palindromic Repeats Gene Editing Genetic Therapy Genetic Vectors
Adenovirus CRISPR Gene delivery Gene therapy Genome editing

Journal

Journal of controlled release : official journal of the Controlled Release Society
ISSN: 1873-4995
Titre abrégé: J Control Release
Pays: Netherlands
ID NLM: 8607908

Informations de publication

Date de publication:
10 11 2020
Historique:
received: 02 07 2020
revised: 31 08 2020
accepted: 01 09 2020
pubmed: 7 9 2020
medline: 22 6 2021
entrez: 6 9 2020
Statut: ppublish

Résumé

Harnessing the bacterial clustered regularly interspaced short palindromic repeats (CRISPR) system for genome editing in eukaryotes has revolutionized basic biomedical research and translational sciences. The ability to create targeted alterations of the genome through this easy to design system has presented unprecedented opportunities to treat inherited disorders and other diseases such as cancer through gene therapy. A major hurdle is the lack of an efficient and safe in vivo delivery system, limiting most of the current gene therapy efforts to ex vivo editing of extracted cells. Here we discuss the unique features of adenoviral vectors that enable tissue specific and efficient delivery of the CRISPR-Cas machinery for in vivo genome editing.

Identifiants

DOI: 10.1016/j.jconrel.2020.09.003 PMID: 32891680 PMC: PMC8091654
pubmed: 32891680
pii: S0168-3659(20)30511-3
doi: 10.1016/j.jconrel.2020.09.003
pmc: PMC8091654
mid: NIHMS1694288
pii:
doi:

Types de publication

Journal Article Research Support, N.I.H., Extramural Review

Langues

eng

Sous-ensembles de citation

IM

Pagination

788-800

Subventions

Organisme : NCI NIH HHS
ID : R01 CA211096
Pays : United States
Organisme : NIBIB NIH HHS
ID : R01 EB026468
Pays : United States
Organisme : NHLBI NIH HHS
ID : T32 HL007088
Pays : United States
Organisme : NCATS NIH HHS
ID : UG3 TR002851
Pays : United States

Informations de copyright

Copyright © 2020 Elsevier B.V. All rights reserved.

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Auteurs

Paul Boucher (P)

Department of Biomedical Engineering, McKelvey School of Engineering, Washington University in Saint Louis, St. Louis, MO 63130, USA; Division of Cancer Biology, Department of Radiation Oncology, School of Medicine, Washington University in Saint Louis, St. Louis, MO 63110, USA.

Xiaoxia Cui (X)

Genome Engineering & iPSC Center, Department of Genetics, School of Medicine, Washington University in Saint Louis, St. Louis, MO 63110, USA.

David T Curiel (DT)

Department of Biomedical Engineering, McKelvey School of Engineering, Washington University in Saint Louis, St. Louis, MO 63130, USA; Division of Cancer Biology, Department of Radiation Oncology, School of Medicine, Washington University in Saint Louis, St. Louis, MO 63110, USA; Biologic Therapeutics Center, Department of Radiation Oncology, School of Medicine, Washington University in Saint Louis, St. Louis, MO 63110, USA. Electronic address: dcuriel@wustl.edu.

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Classifications MeSH

questionsmedicales.fr › Phénomènes génétiques › Régulation de l'expression des gènes › Épigenèse génétique › Extinction de l'expression des gènes › Systèmes CRISPR-Cas › Adenoviral vectors for in vivo delivery of...
questionsmedicales.fr › Phénomènes génétiques › Structures génétiques › Génome › Composants de génome › Séquences répétées en tandem › Clustered regularly interspaced short palindromic repeats › Adenoviral vectors for in vivo delivery of...
questionsmedicales.fr › Techniques d'investigation › Techniques génétiques › Génie génétique › Édition de gène › Adenoviral vectors for in vivo delivery of...
questionsmedicales.fr › Techniques d'investigation › Techniques génétiques › Génie génétique › Thérapie génétique › Adenoviral vectors for in vivo delivery of...
questionsmedicales.fr › Phénomènes génétiques › Structures génétiques › Vecteurs génétiques › Adenoviral vectors for in vivo delivery of...