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Gene knockdown using micro RNA (miRNA)-based vector constructs is likely to become a prominent gene therapy approach. It was the aim of this study to improve the efficiency of gene knockdown through optimizing the structure of miRNA mimics. Knockdown of two target genes was analyzed: CCR5 and green fluorescent protein. We describe here a novel and optimized(More)
Lentiviral vectors are now widely considered one of the safest and most efficient tools for gene delivery and stable gene expression. Even though inducible gene expression cassettes are mandatory for many genetic engineering strategies, most current systems suffer from various issues, such as the requirement of two vectors, which decreases the overall(More)
Pluripotent stem cell (PSC)-based cell therapy is an attractive concept for neurodegenerative diseases, but can lead to tumor formation. This is particularly relevant as proliferating neural precursors rather than postmitotic mature neurons need to be transplanted. Thus, safety mechanisms to eliminate proliferating cells are needed. Here, we propose a(More)
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