Automated Good Manufacturing Practice-Compatible CRISPR-Cas9 Editing of Hematopoietic Stem and Progenitor Cells for Clinical Treatment of β-Hemoglobinopathies.
Journal
The CRISPR journal
ISSN: 2573-1602
Titre abrégé: CRISPR J
Pays: United States
ID NLM: 101738191
Informations de publication
Date de publication:
02 2023
02 2023
Historique:
pubmed:
21
1
2023
medline:
17
2
2023
entrez:
20
1
2023
Statut:
ppublish
Résumé
Cellular therapies hold enormous potential for the cure of severe hematological and oncological disorders. The forefront of innovative gene therapy approaches including therapeutic gene editing and hematopoietic stem cell transplantation needs to be processed by good manufacturing practice to ensure safe application in patients. In the present study, an effective transfection protocol for automated clinical-scale production of genetically modified hematopoietic stem and progenitor cells (HSPCs) using the CliniMACS Prodigy
Identifiants
pubmed: 36662546
doi: 10.1089/crispr.2022.0086
pmc: PMC9986018
doi:
Types de publication
Journal Article
Research Support, Non-U.S. Gov't
Langues
eng
Sous-ensembles de citation
IM
Pagination
5-16Références
Front Genome Ed. 2021 Aug 02;3:618111
pubmed: 34713248
Proc Natl Acad Sci U S A. 2009 Oct 13;106(41):17413-8
pubmed: 19805084
N Engl J Med. 2021 Jan 21;384(3):252-260
pubmed: 33283989
Nature. 2016 Nov 17;539(7629):384-389
pubmed: 27820943
J Clin Invest. 2013 Jul 1;:
pubmed: 23863630
Trends Biotechnol. 2018 Feb;36(2):173-185
pubmed: 29305085
Blood. 2012 Oct 11;120(15):2945-53
pubmed: 22904296
Blood. 2016 Mar 17;127(11):1481-92
pubmed: 26679864
Sci Rep. 2018 Feb 1;8(1):2080
pubmed: 29391533
Haematologica. 2019 May;104(5):881-893
pubmed: 30923099
Cell Res. 2017 Jan;27(1):154-157
pubmed: 27910851
Cells. 2021 Apr 26;10(5):
pubmed: 33925968
Curr Stem Cell Rep. 2022;8(2):72-92
pubmed: 35502223
Transfus Med Hemother. 2019 Jun;46(3):188-196
pubmed: 31244587
Bone Marrow Transplant. 2019 Dec;54(12):1940-1950
pubmed: 30903024
J Biotechnol. 2015 Aug 20;208:44-53
pubmed: 26003884
Front Med (Lausanne). 2020 Nov 26;7:592242
pubmed: 33324662
Drug Deliv. 2018 Nov;25(1):1234-1257
pubmed: 29801422
Blood. 2013 Apr 18;121(16):3246-53
pubmed: 23422750
J Vis Exp. 2018 Apr 10;(134):
pubmed: 29708546
Sci Rep. 2020 Jun 23;10(1):10133
pubmed: 32576837
Biol Blood Marrow Transplant. 2014 Mar;20(3):295-308
pubmed: 24141007
Cytotherapy. 2020 Jul;22(7):388-397
pubmed: 32414635
Cell Stem Cell. 2014 Nov 6;15(5):643-52
pubmed: 25517468
Biol Blood Marrow Transplant. 2020 Jul;26(7):e145-e146
pubmed: 32305358