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Ex vivo gene therapy is an interesting alternative to orthotopic liver transplantation (OLT) for treating metabolic liver diseases. In this study, we investigated its efficacy and biosafety in nonhuman primates. Hepatocytes isolated from liver lobectomy we ...
Recombinant protein-based biologics are increasingly relevant in the pharmaceutical industry. Currently, mammalian cell-based bioprocesses are a routine manufacturing procedure for complex proteins such as recombinant antibodies, and the number of new biol ...
Collinear expression of 5′ Hoxd genes during mammalian limb development is required to properly determine digits number and identity. We recently proposed a two-step model to account for the underlying regulatory mechanism, which involves an initial loopin ...
X-linked adrenoleukodystrophy (X-ALD) is the most common inherited peroxisomal disorder characterized by a progressive demyelination of the central nervous system. The marked loss of myelin and oligodendrocytes observed in the disease prompted us to evalua ...
The diagnosis of muscular dystrophies or the assessment of the functional benefit of gene or cell therapies can be difficult, especially for poorly accessible muscles, and it often lacks a single-fiber resolution. In the present study, we evaluated whether ...
Forkhead/winged-helix transcription factors are evolutionarily conserved and found in organisms as diverse as yeast and humans. They regulate a broad spectrum of events during embryonic and post natal development, including cell cycle regulation, survival, ...
In the past, microinjection of plasmid DNA into early embryos represented the state of the art to generate transgenic zebrafish. However, this approach suffers significant drawbacks (mosaic distribution of the injected transgene, late transgene integration ...
This contribution summarizes first some of our efforts in imaging G-protein-coupled receptor (GPCR) functional inserted into planar tethered lipid bilayer membranes (tBLMs) as a novel platform for biophysical studies. The authors introduced recently a nove ...
Drug-inducible systems allowing the control of transgene expression and knockdown in mammalian cells are invaluable tools for genetic research, and could also play important roles in translational research or gene therapy. We and others have developed a le ...
There is great interest in genetic modification of bone marrow-derived mesenchymal stem cells (MSC), not only for research purposes but also for use in (autologous) patient-derived-patientused transplantations. A major drawback of bulk methods for genetic ...