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- W2531592729 abstract "Gene therapy protocols require robust and long-term gene expression. For two decades, retrovirus family vectors have offered several attractive properties as stable gene-delivery vehicles. These vectors represent a technology with widespread use in basic biology and translational studies that require persistent gene expression for treatment of several monogenic diseases. Immunogenicity and insertional mutagenesis represent the main obstacles to a wider clinical use of these vectors. Efficient and safe non-viral vectors are emerging as a promising alternative and facilitate clinical gene therapy studies. Here, we present an updated review for beginners and expert readers on retro and lentiviruses and the latest generation of transposon vectors (sleeping beauty and piggyBac) used in stable gene transfer and gene therapy clinical trials. We discuss the potential advantages and disadvantages of these systems such as cellular responses (immunogenicity or genome modification of the target cell) following exogenous DNA integration. Additionally, we discuss potential implications of these genome modification tools in gene therapy and other basic and applied science contexts." @default.
- W2531592729 created "2016-10-21" @default.
- W2531592729 creator A5001924735 @default.
- W2531592729 creator A5009132757 @default.
- W2531592729 creator A5033717013 @default.
- W2531592729 creator A5045450377 @default.
- W2531592729 creator A5055870287 @default.
- W2531592729 creator A5061554405 @default.
- W2531592729 date "2016-10-12" @default.
- W2531592729 modified "2023-10-18" @default.
- W2531592729 title "Retroviral vectors and transposons for stable gene therapy: advances, current challenges and perspectives" @default.
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