Characterization of RNA editing and gene therapy with a compact CRISPR-Cas13 in the retina.

Animals Humans CRISPR-Cas Systems Mice Vascular Endothelial Growth Factor A / genetics Genetic Therapy / methods RNA Editing / genetics Retina / metabolism Dependovirus / genetics Mice, Transgenic Retinal Pigment Epithelium / metabolism
CRISPR-Cas13 RNA editing VEGF gene therapy ocular neovascularization

Journal

Proceedings of the National Academy of Sciences of the United States of America
ISSN: 1091-6490
Titre abrégé: Proc Natl Acad Sci U S A
Pays: United States
ID NLM: 7505876

Informations de publication

Date de publication:
05 Nov 2024
Historique:
medline: 31 10 2024
pubmed: 30 10 2024
entrez: 30 10 2024
Statut: ppublish

Résumé

CRISPR-Cas13 nucleases are programmable RNA-targeting effectors that can silence gene expression in a transient manner. Recent iterations of Cas13 nucleases are compact for adeno-associated virus (AAV) delivery to achieve strong and persistent expression of various organs in a safe manner. Here, we report significant transcriptomic signatures of Cas13bt3 expression in retinal cells and show all-in-one AAV gene therapy with Cas13bt3 can effectively silence

Identifiants

DOI: 10.1073/pnas.2408345121 PMID: 39475642
pubmed: 39475642
doi: 10.1073/pnas.2408345121
doi:

Substances chimiques

Vascular Endothelial Growth Factor A 0
VEGFA protein, human 0

Types de publication

Journal Article

Langues

eng

Sous-ensembles de citation

IM

Pagination

e2408345121

Subventions

Organisme : DHAC | National Health and Medical Research Council (NHMRC)
ID : 1185600
Organisme : MOST | National Natural Science Foundation of China (NSFC)
ID : 82201227
Organisme : National Natural Science Foundation of China-Guangdong Joint Fund
ID : 2023A1515011225
Organisme : DHAC | National Health and Medical Research Council (NHMRC)
ID : MRF2008912
Organisme : National Stem Cell Foundation of Australia (NSCFA)
ID : 00339
Organisme : Rebecca L. Cooper Medical Research Foundation
ID : n/a

Déclaration de conflit d'intérêts

Competing interests statement:The authors declare no competing interest.

Auteurs

Satheesh Kumar (S)

Centre for Eye Research Australia, Royal Victorian Eye and Ear Hospital, East Melbourne, VIC 3002, Australia.
Ophthalmology, Department of Surgery, University of Melbourne, East Melbourne, VIC 3002, Australia.
Menzies Institute for Medical Research, University of Tasmania, Hobart, TAS 7000, Australia.
ORCID: 0000-0002-1487-4169

Yi-Wen Hsiao (YW)

Centre for Eye Research Australia, Royal Victorian Eye and Ear Hospital, East Melbourne, VIC 3002, Australia.
ORCID: 0000-0002-9261-0353

Vickie H Y Wong (VHY)

Department of Optometry and Vision Sciences, University of Melbourne, Parkville, VIC 3052, Australia.
ORCID: 0000-0001-9356-7556

Deborah Aubin (D)

School of Medical Sciences, Faculty of Medicine and Health, University of Sydney, Camperdown, NSW 2050, Australia.
Stem Cell Medicine and Stem Cell and Organoid Facility, Children's Medical Research Institute, Faculty of Medicine and Health, University of Sydney, Westmead, NSW 2145, Australia.
ORCID: 0009-0007-4139-1676

Jiang-Hui Wang (JH)

Centre for Eye Research Australia, Royal Victorian Eye and Ear Hospital, East Melbourne, VIC 3002, Australia.

Leszek Lisowski (L)

Translational Vectorology Research Unit, Children's Medical Research Institute, Faculty of Medicine and Health, University of Sydney, Westmead, NSW 2145, Australia.
Laboratory of Molecular Oncology and Innovative Therapies, Military Institute of Medicine - National Research Institute, Warsaw 04-349, Poland.
Australian Genome Therapeutics Centre, Children's Medical Research Institute and Sydney Children's Hospitals Network, Westmead, NSW 2145, Australia.

Elizabeth P Rakoczy (EP)

Centre for Ophthalmology and Visual Science, University of Western Australia, Crawley, WA 6009, Australia.

Fan Li (F)

Eye Center, Zhongshan City People's Hospital, Zhongshan, Guangdong Province 528403, China.

Luis Alarcon-Martinez (L)

Centre for Eye Research Australia, Royal Victorian Eye and Ear Hospital, East Melbourne, VIC 3002, Australia.
Ophthalmology, Department of Surgery, University of Melbourne, East Melbourne, VIC 3002, Australia.
ORCID: 0000-0002-3167-7423

Anai Gonzalez-Cordero (A)

School of Medical Sciences, Faculty of Medicine and Health, University of Sydney, Camperdown, NSW 2050, Australia.
Stem Cell Medicine and Stem Cell and Organoid Facility, Children's Medical Research Institute, Faculty of Medicine and Health, University of Sydney, Westmead, NSW 2145, Australia.
ORCID: 0000-0002-1818-0375

Bang V Bui (BV)

Department of Optometry and Vision Sciences, University of Melbourne, Parkville, VIC 3052, Australia.
ORCID: 0000-0001-7298-1352

Guei-Sheung Liu (GS)

Centre for Eye Research Australia, Royal Victorian Eye and Ear Hospital, East Melbourne, VIC 3002, Australia.
Ophthalmology, Department of Surgery, University of Melbourne, East Melbourne, VIC 3002, Australia.
Menzies Institute for Medical Research, University of Tasmania, Hobart, TAS 7000, Australia.
ORCID: 0000-0003-3379-724X

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Classifications MeSH

questionsmedicales.fr › Eucaryotes › Animaux › Characterization of RNA editing and gene...
questionsmedicales.fr › Eucaryotes › Animaux › Chordés › Vertébrés › Mammifères › Eutheria › Primates › Haplorhini › Catarrhini › Hominidae › Humains › Characterization of RNA editing and gene...
questionsmedicales.fr › Phénomènes génétiques › Régulation de l'expression des gènes › Épigenèse génétique › Extinction de l'expression des gènes › Systèmes CRISPR-Cas › Characterization of RNA editing and gene...
questionsmedicales.fr › Eucaryotes › Animaux › Chordés › Vertébrés › Mammifères › Eutheria › Rodentia › Muridae › Murinae › Souris › Characterization of RNA editing and gene...
questionsmedicales.fr › Facteurs biologiques › Protéines et peptides de signalisation intercellulaire › Protéines angiogéniques › Facteurs de croissance endothéliale vasculaire › Facteur de croissance endothéliale vasculaire de type A › Characterization of RNA editing and gene...
questionsmedicales.fr › Techniques d'investigation › Techniques génétiques › Génie génétique › Thérapie génétique › Characterization of RNA editing and gene...
questionsmedicales.fr › Phénomènes génétiques › Régulation de l'expression des gènes › Maturation post-transcriptionnelle des ARN › Édition des ARN › Characterization of RNA editing and gene...
questionsmedicales.fr › Organes des sens › Oeil › Rétine › Characterization of RNA editing and gene...
questionsmedicales.fr › Virus › Virus à ADN › Parvoviridae › Parvovirinae › Dependovirus › Characterization of RNA editing and gene...
questionsmedicales.fr › Eucaryotes › Animaux › Chordés › Vertébrés › Mammifères › Eutheria › Rodentia › Muridae › Murinae › Souris › Souris transgéniques › Characterization of RNA editing and gene...
questionsmedicales.fr › Organes des sens › Oeil › Rétine › Épithélium pigmentaire de la rétine › Characterization of RNA editing and gene...