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Nicole Déglon

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Publications associées (60)

Progressive and selective striatal degeneration in primary neuronal cultures using lentiviral vector coding for a mutant huntingtin fragment

Diana Zala, Patrick Aebischer, Valérie Gafner, Nicole Déglon

A lentiviral vector expressing a mutant huntingtin protein (htt171-82Q) was used to generate a chronic model of Huntington's disease (HD) in rat primary striatal cultures. In this model, the majority of neurons expressed the transgene so that Western blot ...

2005

Long-term lentiviral-mediated expression of ciliary neurotrophic factor in the striatum of Huntington's disease transgenic mice

Diana Zala, Patrick Aebischer, Jean-Charles Bensadoun, Nicole Déglon

Ciliary neurotrophic factor (CNTF) has been shown to prevent behavioral deficits and striatal degeneration in neurotoxic models of Huntington's disease (HD), but its effect in a genetic model has not been evaluated. Lentiviral vectors expressing the human ...

2004

Encapsulated GDNF-producing C2C12 cells for Parkinson's disease: a pre-clinical study in chronic MPTP-treated baboons

Patrick Aebischer, Nicole Déglon

Glial cell line-derived neurotrophic factor (GDNF), a potent neurotrophic factor with restorative effects in a variety of rodent and primate models of Parkinson's disease (PD), could be of therapeutic value to PD. In this study, we show that intraventricul ...

2004

Local GDNF expression mediated by lentiviral vector protects facial nerve motoneurons but not spinal motoneurons in SOD1(G93A) transgenic mice

Patrick Aebischer, Nicole Déglon, Sandrine Guillot

Approximately 2% of amyotrophic lateral sclerosis (ALS) cases are associated with mutations in the cytosolic Cu/Zn superoxide dismutase 1 (SOD1) gene. Transgenic SOD1 mice constitute useful models of ALS to screen therapeutical approaches. Glial cell line- ...

2004

Neuroprotective gene therapy for Huntington's disease, using polymer-encapsulated cells engineered to secrete human ciliary neurotrophic factor: results of a phase I study

Patrick Aebischer, Nicole Déglon

Huntington's disease (HD) is a monogenic neurodegenerative disease that affects the efferent neurons of the striatum. The protracted evolution of the pathology over 15 to 20 years, after clinical onset in adulthood, underscores the potential of therapeutic ...

2004

Survival of encapsulated human primary fibroblasts and erythropoietin expression under xenogeneic conditions

Patrick Aebischer, Nicole Déglon, Bernard Schneider

Allogeneic cells are the most attractive source for cell transplantation, as the use of xenogeneic cells is hampered by safety concerns and the use of autologous cells involves practical difficulties. The immune rejection of allogeneic cells can be overcom ...

2004

Lentiviral nigral delivery of GDNF does not prevent neurodegeneration in a genetic rat model of Parkinson's disease

Patrick Aebischer, Nicole Déglon, Christophe Lo Bianco

Viral delivery of glial cell line-derived neurotrophic factor (GDNF) currently represents one of the most promising neuroprotective strategies for Parkinson's Disease (PD). However, the effect of this neurotrophic factor has never been tested in the newly ...

2004

Lentiviral-mediated gene transfer to model triplet repeat disorders

Diana Zala, Patrick Aebischer, Nicole Déglon

This chapter describes the potential use of viral-mediated gene transfer in the central nervous system as a new strategy in developing animal models of neurodegenerative diseases. To illustrate the approach, procedures for the production of lentiviral vect ...

2004

Comparative study of GDNF delivery systems for the CNS: polymer rods, encapsulated cells, and lentiviral vectors

Patrick Aebischer, Eric Garrison Fine, Jean-Charles Bensadoun, Nicole Déglon

Glial cell line-derived neurotrophic factor (GDNF) holds great promise for the treatment of Parkinson's disease. In humans, its intracerebroventricular administration leads to limiting side effects. Direct parenchymal delivery using mechanical means, or ce ...

2003

Early and reversible neuropathology induced by tetracycline-regulated lentiviral overexpression of mutant huntingtin in rat striatum

Patrick Aebischer, Valérie Gafner, Nicole Déglon

The ability to overexpress full-length huntingtin or large fragments represents an important challenge to mimic Huntington's pathology and reproduce all stages of the disease in a time frame compatible with rodent life span. In the present study, tetracycl ...

Oxford University Press2003

Presence of Gal-alpha1,3Gal epitope on xenogeneic lines: implications for cellular gene therapy based on the encapsulation technology

Patrick Aebischer, Nicole Déglon

Exposure to human serum induces the lysis of xenogeneic cells through natural antibodies and complement activation. The carbohydrate Galactose-alpha1,3-Galactose (Gal-alpha1,3-Gal) epitope, has been shown to be the principal antigenic determinant on target ...

2003

Optimization of human erythropoietin secretion from MLV-infected human primary fibroblasts used for encapsulated cell therapy

Patrick Aebischer, Nicole Déglon

BACKGROUND: The transplantation of encapsulated cells genetically engineered to secrete human erythropoietin (hEpo) represents an alternative to repeated injections of the recombinant hormone for the treatment of Epo-responsive anemia. In the present study ...

2003

Activity analysis of housekeeping promoters using self-inactivating lentiviral vector delivery into the mouse retina

Patrick Aebischer, Nicole Déglon, Didier Trono

For most retinal degeneration disorders, no efficient treatment exists to preserve photoreceptors (PRs) and, consequently, to maintain vision. Gene transfer appears to be a promising approach to prevent PR loss. In order to design adequate vectors to targe ...

2003

Lentivirally delivered glial cell line-derived neurotrophic factor increases the number of striatal dopaminergic neurons in primate models of nigrostriatal degeneration

Patrick Aebischer, Nicole Déglon

The primate striatum contains tyrosine hydroxylase (TH)-immunoreactive (ir) neurons, the numbers of which are augmented after dopamine depletion. Glial cell line-derived neurotrophic factor (GDNF) strongly modulates the viability and phenotypic expression ...

2002

Long-term doxycycline-regulated secretion of erythropoietin by encapsulated myoblasts

Patrick Aebischer, Nicole Déglon, Bernard Schneider

We developed an ex vivo gene therapy approach for the regulated delivery of therapeutic proteins based on the implantation of encapsulated, genetically engineered C(2)C(12) myoblasts. We investigated doxycycline-based regulation of gene expression to modul ...

2002

alpha -Synucleinopathy and selective dopaminergic neuron loss in a rat lentiviral-based model of Parkinson's disease

Patrick Aebischer, Nicole Déglon, Christophe Lo Bianco, Bernard Schneider

Parkinson's disease (PD) is characterized by the progressive loss of substantia nigra dopaminergic neurons and the presence of cytoplasmic inclusions named Lewy bodies. Two missense mutations of the alpha-synuclein (alpha-syn; A30P and A53T) have been desc ...

National Academy of Sciences2002

Delivery of erythropoietin by encapsulated myoblasts in a genetic model of severe anemia

Patrick Aebischer, Nicole Déglon, Bernard Schneider

BACKGROUND: Existing animal models of anemia inadequately reflect the hematocrit usually present in chronic renal failure (CRF) patients and do not permit long-term treatment studies. The transgenic mouse strain 134.3LC (Epo-TAg(H)) displays a severe chron ...

2002

Seizure suppression by adenosine-releasing cells is independent of seizure frequency

Patrick Aebischer, Nicole Déglon, Vivianne Padrun

PURPOSE: Intraventricular cellular delivery of adenosine was recently shown to be transiently efficient in the suppression of seizure activity in the rat kindling model of epilepsy. We tested whether the suppression of seizures by adenosine-releasing graft ...

2002

Dose-dependent neuroprotective effect of ciliary neurotrophic factor delivered via tetracycline-regulated lentiviral vectors in the quinolinic acid rat model of Huntington's disease

Patrick Aebischer, Nicole Déglon

The ability to regulate gene expression constitutes a prerequisite for the development of gene therapy strategies aimed at the treatment of neurologic disorders. In the present work, we used tetracycline (Tet)-regulated lentiviral vectors to investigate th ...

2002

Neurospheres modified to produce glial cell line-derived neurotrophic factor increase the survival of transplanted dopamine neurons

Patrick Aebischer, Nicole Déglon

Glial cell line-derived neurotrophic factor (GDNF) has been shown to increase the survival of dopamine neurons in a variety of in vitro and in vivo model systems. Therefore, it constitutes an important therapeutic protein with the potential to ameliorate d ...

2002

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