Concept

Gene therapy

Related publications (480)

Virus-free transient protein production in Sf9 cells

Florian Maria Wurm, David Hacker, Lucia Baldi Unser, Xiao Shen

A method for virus-free transient gene expression from suspension-adapted Sf9 insect cells was developed with the gene of interest being expressed from a plasmid carrying the homologous region 5 enhancer (hr5) and the immediate early 1 (ie1) promoter from ...

Elsevier2014

Dual-regulated Lentiviral Vector for Gene Therapy of X-linked Chronic Granulomatosis

Didier Trono

Regulated transgene expression may improve the safety and efficacy of hematopoietic stem cell (HSC) gene therapy. Clinical trials for X-linked chronic granulomatous disease (X-CGD) employing gammaretroviral vectors were limited by insertional oncogenesis o ...

Elsevier2014

Sustained Delivery of Insulin-Like Growth Factor-1/Hepatocyte Growth Factor Stimulates Endogenous Cardiac Repair in the Chronic Infarcted Pig Heart

Maartje Martina Cornelia Bastings

Activation of endogenous cardiac stem/progenitor cells (eCSCs) can improve cardiac repair after acute myocardial infarction. We studied whether the in situ activation of eCSCs by insulin-like growth factor 1 (IGF-1) and hepatocyte growth factor (HGF) could ...

2014

Control of dopaminergic neuron survival by the unfolded protein response transcription factor XBP1

Bernard Schneider, Pamela Solange Valdés Undurraga

Parkinson disease (PD) is characterized by the selective loss of dopaminergic neurons of the substantia nigra pars compacta (SNpc). Although growing evidence indicates that endoplasmic reticulum (ER) stress is a hallmark of PD, its exact contribution to th ...

National Academy of Sciences2014

Modulation of mitophagy and use thereof

Didier Trono, Isabelle Barde

The present invention relates to an agent or composition comprising at least one of: (i) a micro-RNA targeting a mitophagy-associated gene or a variant thereof, (ii) a vector comprising a nucleic acid encoding said micro-RNA or variant thereof, (iii) an in ...

2014

Genetic engineering of hematopoiesis for targeted IFN-α delivery inhibits breast cancer progression

Michele De Palma, Mario Leonardo Squadrito

The immunosuppressive tumor microenvironment represents a major hurdle to cancer therapy. We developed a gene transfer strategy into hematopoietic stem cells (HSCs) to target transgene expression to tumor-infiltrating monocytes/macrophages. Using a combina ...

2014

Derivation of Traceable and Transplantable Photoreceptors from Mouse Embryonic Stem Cells

Freddy Radtke, Ute Koch

Retinal degenerative diseases resulting in the loss of photoreceptors are one of the major causes of blindness. Photoreceptor replacement therapy is a promising treatment because the transplantation of retina-derived photoreceptors can be applied now to di ...

Cell Press2014

A high-capacity cell macroencapsulation system supporting the long-term survival of genetically engineered allogeneic cells

Patrick Aebischer, Matthias Lütolf, Bernard Schneider, Steffen Cosson, Aurélien Lathuilière

The rapid increase in the number of approved therapeutic proteins, including recombinant antibodies, for diseases necessitating chronic treatments raises the question of the overall costs imposed on healthcare systems. It is therefore important to investig ...

Elsevier2014

Parkinson's disease-linked mutations in VPS35 induce dopaminergic neurodegeneration

Bernard Schneider, Liliane Glauser, Elpida Tsika, Darren Moore, Guillaume Daniel, Aris Nikolaos Fiser

Mutations in the vacuolar protein sorting 35 homolog (VPS35) gene at the PARK17 locus, encoding a key component of the retromer complex, were recently identified as a new cause of late-onset, autosomal dominant Parkinson's disease (PD). Here we explore the ...

Oxford University Press2014

Functional polypeptide and hybrid materials: Precision synthesis via alpha-amino acid N-carboxyanhydride polymerization and emerging biomedical applications

Harm-Anton Klok

Polypeptides derived from naturally occurring a-amino acids have emerged as a unique and versatile family of bio-inspired biomaterials that can be tailor-made for varying biomedical applications such as controlled drug release, gene delivery, tissue engine ...

Elsevier2014

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