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

Related publications (34)

Embracing the Modern Biochemistry of Brain Metabolism

This editorial challenges the long‐held neuron‐centered view of brain metabolism, relying on ample evidence that it is a cooperative, multicellular process. Astrocytes, oligodendrocytes, and other glia play active roles providing lactate, antioxidant suppo ...

Wiley2025

Research Priorities on the Role of α-Synuclein in Parkinson's Disease Pathogenesis

Hilal Lashuel

Various forms of Parkinson's disease, including its common sporadic form, are characterized by prominent α-synuclein (αSyn) aggregation in affected brain regions. However, the role of αSyn in the pathogenesis and evolution of the disease remains unclear, d ...

2024

The role of the NF-kappaB pathway in Amyotrophic lateral sclerosis pathogenesis

Emma Charlotta Källstig

Amyotrophic lateral sclerosis (ALS) is a neurodegenerative motor disorder, which results in death within a few years of diagnosis. While the cause of most cases of ALS is unknown, 10% of cases are familial (fALS), and associated with mutations in one of ov ...

EPFL2023

Paradoxical neuronal hyperexcitability in a mouse model of mitochondrial pyruvate import deficiency

Maria del Carmen Sandi Perez, Simone Astori, Eva Ramos Fernandez

Neuronal excitation imposes a high demand of ATP in neurons. Most of the ATP derives primarily from pyruvate-mediated oxidative phosphorylation, a process that relies on import of pyruvate into mitochondria occuring exclusively via the mitochondrial pyruva ...

eLIFE SCIENCES PUBL LTD2022

Core-shell Structured Chitosan-polyethylenimine Nanoparticles for Gene Delivery: Improved Stability, Cellular Uptake, and Transfection Efficiency

Sandrine Gerber, Perrine Agnes Edith Robin, Laura Camille Louise Nicolle

Gene therapy emerged as a promising treatment option for acquired and inherited diseases. The delivery of nucleic acids relies on vectors that condense and encapsulate their cargo. Especially non-viral gene delivery systems are of increasing interest. Howe ...

2022

Engineering synthetic adhesins for biophysical investigation and rewiring of host-microbe interactions

Xavier Jean-Yves Pierrat

Host attachment is often a critical step in the onset of pathogenesis. To attach to host cells, bacteria have evolved a range of adhesins that bind to specific receptors. Some of these adhesins have been thoroughly characterized using biochemical technique ...

EPFL2022

Engineering of chitosan-based derivatives for non-viral gene delivery

Laura Camille Louise Nicolle

Gene therapy offers the possibility to treat or even cure diseases originating from genetic defects by introducing a therapeutic gene in target cells or correcting the initial defective gene. Amongst different options, non-viral vectors rely on the deliver ...

EPFL2022

A conditional glutamatergic synaptic vesicle marker for Drosophila

Evelyne Ruchti, Brian Donal McCabe

Glutamate is a principal neurotransmitter used extensively by the nervous systems of all vertebrate and invertebrate animals. It is primarily an excitatory neurotransmitter that has been implicated in nervous system development, as well as a myriad of brai ...

OXFORD UNIV PRESS INC2022

A cell fitness selection model for neuronal survival during development

Gioele La Manno

Developmental cell death plays an important role in the construction of functional neural circuits. In vertebrates, the canonical view proposes a selection of the surviving neurons through stochastic competition for target-derived neurotrophic signals, imp ...

Nature Research2019

The virtuous cycle of human genetics and mouse models in drug discovery

Johan Auwerx

Ongoing studies in many species seek to understand the origins, architecture and consequences of phenotypic variation under normal and dysfunctional conditions, with the aim of identifying targets for intervention that can prevent, stabilize or reverse dis ...

NATURE PUBLISHING GROUP2019

A microfluidic array for high-content screening at whole-organism resolution

Martinus Adela Maria Gijs, Johan Auwerx, Matteo Cornaglia, Laurent Mouchiroud, Daniel Migliozzi

A main step for the development and the validation of medical drugs is the screening on whole organisms, which gives the systemic information that is missing when using cellular models. Among the organisms of choice, Caenorhabditis elegans is a soil worm w ...

SPIE-INT SOC OPTICAL ENGINEERING2018

Development of Covalent Chitosan-Polyethylenimine Derivatives as Gene Delivery Vehicle: Synthesis, Characterization, and Evaluation

Sandrine Gerber, Céline Marie Anne Journot, Laura Camille Louise Nicolle

There is an increasing interest in cationic polymers as important constituents of non-viral gene delivery vectors. In the present study, we developed a versatile synthetic route for the production of covalent polymeric conjugates consisting of water-solubl ...

2021

Data-driven reconstruction of a point neuron mouse brain

Csaba Erö

In this work, we present a semi-automatic method to reconstruct a mouse whole-brain model at the point-neuron level by integrating a wide array of biological data. Our process has three parts: cell position and type assignment, connectivity mapping, and si ...

EPFL2018

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

Modeling spinal locomotor circuits for movements in developing zebrafish

Yann Roussel

Many spinal circuits dedicated to locomotor control have been identified in the developing zebrafish. How these circuits operate together to generate the various swimming movements during development remains to be clarified. In this study, we iteratively b ...

eLIFE SCIENCES PUBL LTD2021

Variants in USP48 encoding ubiquitin hydrolase are associated with autosomal dominant non-syndromic hereditary hearing loss

Nicolas Jean Philippe Guex

Non-Syndromic Hereditary Hearing Loss (NSHHL) is a genetically heterogeneous sensory disorder with about 120 genes already associated. Through exome sequencing (ES) and data aggregation, we identified a family with six affected individuals and one unrelate ...

Oxford University Press2021

MAR-Mediated transgene integration into permissive chromatin and increased expression by recombination pathway engineering

Christian Iseli, Nicolas Mermod, Thomas Junier

Untargeted plasmid integration into mammalian cell genomes remains a poorly understood and inefficient process. The formation of plasmid concatemers and their genomic integration has been ascribed either to non-homologous end-joining (NHEJ) or homologous r ...

2017

WDR79/TCAB1 plays a conserved role in the control of locomotion and ameliorates phenotypic defects in SMA models

Brian Donal McCabe

SMN (Survival Motor Neuron) deficiency is the predominant cause of spinal muscular atrophy (SMA), a severe neurodegenerative disorder that can lead to progressive paralysis and death. Although SMN is required in every cell for proper RNA metabolism, the re ...

Academic Press Inc Elsevier Science2017

Parkinson mice show functional and molecular changes in the gut long before motoric disease onset

Hilal Lashuel

Background: There is increasing evidence that Parkinson's disease (PD) might start in the gut, thus involving and compromising also the enteric nervous system (ENS). At the clinical onset of the disease the majority of dopaminergic neurons in the midbrain ...

2021

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