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Identification of genetic insulator elements to increase the safety of viral gene therapy vectors

Related publications (77)

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Intracerebroventricular Injection of Adeno-Associated Virus 6 and 9 Vectors for Cell Type-Specific Transgene Expression in the Spinal Cord

Patrick Aebischer, Bernard Schneider, Christopher Towne, Elisabeth Anne Dirren, Veronica Setola

In the context of motoneuron diseases, gene delivery as an experimental or therapeutic approach is hindered by the challenge to specifically target cell populations that are widely distributed along the spinal cord. Further complicating the task, transgene ...

Mary Ann Liebert2014

MAR-mediated integration of plasmid vectors for in vivo gene transfer and regulation

Andrzej Kulik, Nicolas Mermod, Stefania Puttini, Déborah Ley, Damien Saugy

Background: The in vivo transfer of naked plasmid DNA into organs such as muscles is commonly used to assess the expression of prophylactic or therapeutic genes in animal disease models. Results: In this study, we devised vectors allowing a tight regulatio ...

BioMed Central2013

MAR Elements and Transposons for Improved Transgene Integration and Expression

Nicolas Mermod, Déborah Ley, Niamh Harraghy

Reliable and long-term expression of transgenes remain significant challenges for gene therapy and biotechnology applications, especially when antibiotic selection procedures are not applicable. In this context, transposons represent attractive gene transf ...

2013

Gene therapy: too much splice can spoil the dish

Didier Trono

The use of integrating vectors for gene therapy - required for stable correction of gene expression - carries the risk of insertional mutagenesis, which can lead to activation of a tumorigenic program. In this issue of the JCI, Moiani et al. and Cesana et ...

2012

Parkinson's Disease: Gene Therapies

Patrick Aebischer, Bernard Schneider, Philippe Guillaume Coune

With the recent development of effective gene delivery systems, gene therapy for the central nervous system is finding novel applications. Here, we review existing viral vectors and discuss gene therapy strategies that have been proposed for Parkinson's di ...

Cold Spring Harbor Lab Press, Publications Dept2012

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Insertional mutagenesis represents a serious adverse effect of gene therapy with integrating vectors. However, although uncontrolled activation of growth-promoting genes in stem cells can predictably lead to oncological processes, this is far less likely i ...

2011

The delivery of exogenous nucleic acids into mammalian cells is a valuable technique for basic research studies on the expression, regulation and function of genes and proteins. At the same time, gene delivery to cultivated mammalian cells has become funda ...

EPFL2011

In vivo lentiviral vector (LV)-mediated gene delivery would represent a great step forward in the field of gene therapy. Therefore, we have engineered a novel LV displaying SCF and a mutant cat endogenous retroviral glycoprotein, RDTR. These RDTR/SCF-LVs o ...

2011

Lentiviral Vector-Mediated Gene Delivery for the Generation of Chinese Hamster Ovary Cell Lines with Improved Productivity and Stability

Agata Oberbek

Recombinant cell line generation by standard transfection techniques is a time-consuming and labor-intensive process often leading to an unpredictable outcome as transgene integration is a rare, random event. Consequently, the population of cells obtained ...

EPFL2010

The Interplay between Cytidine Deaminases, HIV and Domesticated Genetic Parasites

"There is more virus in us than us in us". John Coffin's famous sentence illustrates that particular nucleic acid sequences related to exogenous viruses, called retrotransposons, constitute almost half of the human genome and largely exceeds the amount of ...

EPFL2010