Validation of Gene Therapy for Mutant Mitochondria by Delivering Mitochondrial RNA Using a MITO-Porter.
MITO-Porter
heteroplasmic mutation
mitochondrial delivery
mitochondrial gene therapy
nucleic acids medicine
Journal
Molecular therapy. Nucleic acids
ISSN: 2162-2531
Titre abrégé: Mol Ther Nucleic Acids
Pays: United States
ID NLM: 101581621
Informations de publication
Date de publication:
05 Jun 2020
05 Jun 2020
Historique:
received:
24
02
2020
revised:
10
04
2020
accepted:
15
04
2020
pubmed:
11
5
2020
medline:
11
5
2020
entrez:
11
5
2020
Statut:
ppublish
Résumé
Here, we report on validating a mitochondrial gene therapy by delivering nucleic acids to mitochondria of diseased cells by a MITO-Porter, a liposome-based carrier for mitochondrial delivery. We used cells derived from a patient with a mitochondrial disease with a G625A heteroplasmic mutation in the tRNA
Identifiants
pubmed: 32388194
pii: S2162-2531(20)30112-8
doi: 10.1016/j.omtn.2020.04.004
pmc: PMC7210581
pii:
doi:
Types de publication
Journal Article
Langues
eng
Pagination
687-698Informations de copyright
Copyright © 2020 The Authors. Published by Elsevier Inc. All rights reserved.
Références
Bioconjug Chem. 2001 Nov-Dec;12(6):1005-11
pubmed: 11716693
Proc Natl Acad Sci U S A. 2012 Mar 27;109(13):4840-5
pubmed: 22411789
Gene Ther. 1998 Jul;5(7):930-7
pubmed: 9813664
Nucleic Acids Res. 1989 Apr 11;17(7):2503-16
pubmed: 2785681
J Control Release. 2018 Mar 28;274:109-117
pubmed: 29408532
Cell Cycle. 2007 Oct 15;6(20):2473-7
pubmed: 17704646
J Mol Cell Cardiol. 2016 Nov;100:43-53
pubmed: 27677939
Annu Rev Biochem. 2011;80:1033-53
pubmed: 21417719
Lancet. 2012 May 12;379(9828):1825-34
pubmed: 22482939
Nanoscale. 2017 Dec 7;9(47):18690-18698
pubmed: 29165486
Mol Ther. 2011 Aug;19(8):1449-56
pubmed: 21694702
Cell. 2010 Aug 6;142(3):362-3
pubmed: 20691896
J Control Release. 2018 Jan 10;269:177-188
pubmed: 29146241
Nat Rev Genet. 2015 Sep;16(9):530-42
pubmed: 26281784
Adv Drug Deliv Rev. 2008 Oct-Nov;60(13-14):1439-62
pubmed: 18655816
J Cell Biol. 1986 Jan;102(1):97-103
pubmed: 2867101
Biomaterials. 2012 Feb;33(5):1589-95
pubmed: 22105068
Macromol Biosci. 2017 Jan;17(1):
pubmed: 27797146
Nucleic Acids Res. 2011 Oct;39(18):8173-86
pubmed: 21724600
Nat Protoc. 2008;3(6):965-76
pubmed: 18536644
Biochim Biophys Acta. 2008 Feb;1778(2):423-32
pubmed: 18054323
J Control Release. 2016 Dec 28;244(Pt B):194-204
pubmed: 27374187
J Cell Sci. 2004 Jun 1;117(Pt 13):2653-62
pubmed: 15138283
Biochem Biophys Res Commun. 2019 Jun 4;513(3):708-713
pubmed: 30987827
Trends Biochem Sci. 2008 Jul;33(7):320-9
pubmed: 18513973
J Laryngol Otol. 2011 Dec;125(12):1282-5
pubmed: 21914246
J Biol Chem. 2006 Feb 10;281(6):3544-51
pubmed: 16326716
Biochemistry. 1996 Jan 23;35(3):1027-36
pubmed: 8547238
Cell. 2011 Aug 19;146(4):645-58
pubmed: 21854988
Biochim Biophys Acta. 2006 Aug;1763(8):833-43
pubmed: 16839621
J Control Release. 2015 Sep 10;213:86-95
pubmed: 26160304