Novel lentiviral vectors for gene therapy of sickle cell disease combining gene addition and gene silencing strategies.
MT: Non-coding RNAs
gene addition
gene silencing
gene therapy
lentiviral vectors
microRNA
sickle cell disease
βs silencing
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:
13 Jun 2023
13 Jun 2023
Historique:
received:
30
07
2022
accepted:
17
03
2023
medline:
24
4
2023
pubmed:
24
4
2023
entrez:
24
04
2023
Statut:
epublish
Résumé
Sickle cell disease (SCD) is due to a mutation in the β-globin gene causing production of the toxic sickle hemoglobin (HbS; α
Identifiants
pubmed: 37090420
doi: 10.1016/j.omtn.2023.03.012
pii: S2162-2531(23)00071-9
pmc: PMC10113710
doi:
Types de publication
Journal Article
Langues
eng
Pagination
229-246Informations de copyright
© 2023 The Authors.
Déclaration de conflit d'intérêts
M.B., F.M., M.C., M.A., and A.M. are the inventors of two patents describing bifunctional LVs for hemoglobinopathies.
Références
Blood. 1984 Apr;63(4):921-6
pubmed: 6200161
Bioinformatics. 2014 Apr 1;30(7):923-30
pubmed: 24227677
PLoS One. 2012;7(10):e47175
pubmed: 23071749
Blood. 2019 Oct 10;134(15):1203-1213
pubmed: 31467062
N Engl J Med. 2022 Feb 17;386(7):617-628
pubmed: 34898139
Lancet. 2017 Jul 15;390(10091):311-323
pubmed: 28159390
Hum Gene Ther. 2019 Jun;30(6):753-761
pubmed: 30700149
J RNAi Gene Silencing. 2011;7:434-42
pubmed: 21769296
Mol Ther Methods Clin Dev. 2020 Mar 17;17:589-600
pubmed: 32300607
Blood Adv. 2020 May 12;4(9):2058-2063
pubmed: 32396618
Blood. 2017 Sep 28;130(13):1597-1599
pubmed: 28830890
Blood. 2014 Jan 23;123(4):481-5
pubmed: 24222332
Nucleic Acids Res. 2019 Jan 8;47(D1):D155-D162
pubmed: 30423142
Biomaterials. 2017 Sep;139:102-115
pubmed: 28599149
Mol Ther. 2022 Jan 5;30(1):145-163
pubmed: 34418541
Ann Hematol. 2008 May;87(5):375-9
pubmed: 18026953
Blood Cells Mol Dis. 2018 May;70:54-65
pubmed: 28651846
Proc Natl Acad Sci U S A. 2008 Apr 15;105(15):5868-73
pubmed: 18398004
Silence. 2011 Oct 26;2:8
pubmed: 22029761
Mol Ther. 2015 Sep;23(9):1465-74
pubmed: 26080908
N Engl J Med. 2021 Jan 21;384(3):252-260
pubmed: 33283989
PLoS One. 2014 Mar 26;9(3):e92188
pubmed: 24670775
PLoS One. 2013;8(3):e59890
pubmed: 23533656
N Engl J Med. 2017 Apr 20;376(16):1561-1573
pubmed: 28423290
Nat Med. 2022 Jan;28(1):81-88
pubmed: 35075288
Sci Adv. 2020 Feb 12;6(7):
pubmed: 32917636
J R Soc Med. 2016 Aug;109(8):303-9
pubmed: 27325377
N Engl J Med. 2017 Mar 2;376(9):848-855
pubmed: 28249145
Mol Ther Methods Clin Dev. 2018 Aug 04;10:268-280
pubmed: 30140714
Nat Med. 2019 Feb;25(2):234-241
pubmed: 30664781
N Engl J Med. 2021 Jan 21;384(3):205-215
pubmed: 33283990
Cell. 2018 Apr 5;173(2):430-442.e17
pubmed: 29606353
Mol Ther. 2021 Sep 1;29(9):2841-2853
pubmed: 33940155
Blood. 2018 Apr 26;131(17):1960-1973
pubmed: 29519807
Nature. 2015 Nov 12;527(7577):192-7
pubmed: 26375006
Mol Ther. 2022 Aug 3;30(8):2693-2708
pubmed: 35526095
N Engl J Med. 2022 Feb 3;386(5):415-427
pubmed: 34891223
Haematologica. 2021 Oct 01;106(10):2740-2745
pubmed: 34047176
Haematologica. 2014 Nov;99(11):1677-85
pubmed: 25107887
Nature. 2010 Sep 16;467(7313):318-22
pubmed: 20844535
Nat Rev Drug Discov. 2019 Jun;18(6):447-462
pubmed: 30858502
Bioinformatics. 2010 Jan 1;26(1):139-40
pubmed: 19910308
Blood. 2021 Mar 18;137(11):1553-1555
pubmed: 33512468
Mol Ther. 2009 Jan;17(1):169-75
pubmed: 19002161
J Biol Chem. 2015 Sep 25;290(39):23523-7
pubmed: 26296885
Blood. 2016 Aug 25;128(8):1139-43
pubmed: 27405777
Nat Biotechnol. 2022 Dec;40(12):1807-1813
pubmed: 35773341
J Clin Invest. 2016 Oct 3;126(10):3868-3878
pubmed: 27599293
Bioinformatics. 2013 Jan 1;29(1):15-21
pubmed: 23104886
N Engl J Med. 2022 Jan 13;386(2):138-147
pubmed: 34898140
Blood. 2011 Jul 7;118(1):19-27
pubmed: 21490337
Arch Intern Med. 1983 May;143(5):1030-2
pubmed: 6679216
Nat Biotechnol. 2006 Jan;24(1):89-94
pubmed: 16378095
CRISPR J. 2022 Feb;5(1):19-30
pubmed: 35099280
Int J Mol Sci. 2021 Mar 31;22(7):
pubmed: 33807258
Mol Ther. 2009 Jun;17(6):1039-52
pubmed: 19293777
Nucleic Acids Res. 2007;35(15):5154-64
pubmed: 17660190
Proc Natl Acad Sci U S A. 2006 Apr 11;103(15):5953-8
pubmed: 16585504
Mol Ther Nucleic Acids. 2020 Mar 6;19:572-580
pubmed: 31927330
Mol Ther Methods Clin Dev. 2018 Mar 10;9:257-269
pubmed: 29707600
Gene Ther. 2022 Sep;29(9):536-543
pubmed: 35194185
Nucleic Acids Res. 1998 Feb 1;26(3):721-9
pubmed: 9443963