Open AccessSustained Knockdown of a Disease‐Causing Gene in Patient‐Specific Induced Pluripotent Stem Cells Using Lentiviral Vector‐Based Gene Therapy
Author(s) -
Eggenschwiler Reto,
Loya Komal,
Wu Guangming,
Sharma Amar Deep,
Sgodda Malte,
Zychlinski Daniela,
Herr Christian,
Steinemann Doris,
Teckman Jeffrey,
Bals Robert,
Ott Michael,
Schambach Axel,
Schöler Hans Robert,
Cantz Tobias
Publication year - 2013
Publication title -
stem cells translational medicine
Language(s) - English
Resource type - Journals
SCImago Journal Rank - 1.781
H-Index - 71
eISSN - 2157-6580
pISSN - 2157-6564
DOI - 10.5966/sctm.2013-0017
Subject(s) - gene knockdown , induced pluripotent stem cell , small hairpin rna , biology , rna interference , gene silencing , viral vector , genetic enhancement , microbiology and biotechnology , cancer research , embryonic stem cell , gene , genetics , rna , recombinant dna
This study proposes an approach for the expression of a therapeutic short hairpin RNA (shRNA) in disease‐specific induced pluripotent stem cells (iPSCs) using third‐generation lentiviral vectors. It was found that lentiviral vector‐mediated expression of shRNAs can be efficiently used to knock down and functionally evaluate disease‐related genes in patient‐specific iPSCs.