Publications de Cylia Cloé Rochat

Astrocyte-targeting RNA interference against mutated superoxide dismutase 1 induces motoneuron plasticity and protects fast-fatigable motor units in a mouse model of amyotrophic lateral sclerosis

Patrick Aebischer, Bernard Schneider, Cylia Cloé Rochat, Nathalie Marie Géraldine Bernard

In amyotrophic lateral sclerosis (ALS) caused by SOD1 gene mutations, both cell-autonomous and noncell-autonomous mechanisms lead to the selective degeneration of motoneurons (MN). Here, we evaluate the therapeutic potential of gene therapy targeting mutat ...

WILEY2022

Altered interplay between endoplasmic reticulum and mitochondria in Charcot-Marie-Tooth type 2A neuropathy

Bernard Schneider, Cylia Cloé Rochat, Mary-Claude Croisier-Coeytaux, Manisha Juneja, Omar El Mansour, Catherine Maclachlan, Roman Chrast, Nathalie Marie Géraldine Bernard

Mutations in the MFN2 gene encoding Mitofusin 2 lead to the development of Charcot-Marie-Tooth type 2A (CMT2A), a dominant axonal form of peripheral neuropathy. Mitofusin 2 is localized at both the outer membrane of mitochondria and the endoplasmic reticul ...

National Academy of Sciences2019

An miRNA-based gene therapy approach to target mutated SOD1 in key cell types in amyotrophic lateral sclerosis (ALS)

Bernard Schneider, Cylia Cloé Rochat, Julianne Bobela

MARY ANN LIEBERT, INC2018

Bicistronic aav vector for rna interference in als

Bernard Schneider, Cylia Cloé Rochat, Julianne Bobela

The present invention relates to a bicistronic expression vector for silencing a gene specifically in astrocytes and neurons, comprising two expression cassettes comprising a first and a second silencer sequence, respectively, wherein the expression of sai ...

2017

AAV based gene therapy for rare diseases : targeting astrocytes for functional recovery in Amyotrophic Lateral Sclerosis

Cylia Cloé Rochat

Of the seven thousand diseases that are described as rare, 80% of them have an identified genetic cause. With this in the mind, the development of technologies, such as gene therapy, to address the genetic factors involved in these pathologies, might be a ...

EPFL2017

Use of a Bicistronic Vector to Silence SOD1 in Motoneurons and Astrocytes for the Treatment of Familial Amyotrophic Lateral Sclerosis

Patrick Aebischer, Bernard Schneider, Cylia Cloé Rochat, Julianne Bobela, Nathalie Marie Géraldine Bernard

Nature Publishing Group2016

Characterization Of Motor And Sensory Neuronal Dysfunction In Both In Vitro And In Vivo Models Of Cmt2A Pathology

Bernard Schneider, Cylia Cloé Rochat, Roman Chrast, Nathalie Marie Géraldine Bernard

Wiley-Blackwell2016

Tmc gene therapy restores auditory function in deaf mice

Patrick Aebischer, Bernard Schneider, Cylia Cloé Rochat

Genetic hearing loss accounts for up to 50% of prelingual deafness worldwide, yet there are no biologic treatments currently available. To investigate gene therapy as a potential biologic strategy for restoration of auditory function in patients with genet ...

American Association for the Advancement of Science2015

SOD1 silencing in motoneurons or glia rescues neuromuscular function in ALS mice

Patrick Aebischer, Bernard Schneider, Cylia Cloé Rochat, Christopher Towne, Julianne Bobela, Elisabeth Anne Dirren

Objective: Amyotrophic lateral sclerosis is an incurable disorder mainly characterized by motoneuron degeneration. Mutations in the superoxide dismutase 1 (SOD1) gene account for 20% of familial forms of the disease. Mutant SOD1 exerts multiple pathogenic ...

Wiley-Blackwell2015