Publications related to Lentiviral and Adeno-Associated Vector-Based Therapy for Motor Neuron Disease Through RNAi

Related publications (32)

Targeting TDP-43 Proteinopathies in Neurodegeneration by its Native-State Stabilization

Preventing the misfolding or aggregation of TDP-43 is the most actively pursued disease-modifying strategy to treat amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases. In our work, we provide proof of concept that native state stabili ...

EPFL2024

Trio preserves motor synapses and prolongs motor ability during aging

Evelyne Ruchti, Brian Donal McCabe, Wei Jiao, Soumya Banerjee, Greta Limoni, Samuel William Vernon, Marine Van Campenhoudt

The decline of motor ability is a hallmark feature of aging and is accompanied by degeneration of motor synaptic terminals. Consistent with this, Drosophila motor synapses undergo characteristic age -dependent structural fragmentation co -incident with dim ...

Cell Press2024

The role of LRRK2 in lung adenocarcinoma

Aspasia Gkasti

Lung cancer is the leading cause of cancer-related deaths worldwide and the most common lung cancer subtype is lung adenocarcinoma (LUAD). Frequently mutated genes involve activating mutations in KRAS and loss of function mutations in TP53. LUADs primarily ...

EPFL2024

Inheritance of H3K9 methylation regulates genome architecture in Drosophila early embryos

Fides Zenk

Constitutive heterochromatin is essential for transcriptional silencing and genome integrity. The establishment of constitutive heterochromatin in early embryos and its role in early fruitfly development are unknown. Lysine 9 trimethylation of histone H3 ( ...

Springernature2024

Convalescent human plasma candidate reference materials protect against Crimean-Congo haemorrhagic fever virus (CCHFV) challenge in an A129 mouse model

Frank William Charlton

Crimean-Congo Haemorrhagic Fever Virus (CCHFV) is spread by infected ticks or direct contact with blood, tissues and fluids from infected patients or livestock. Infection with CCHFV causes severe haemorrhagic fever in humans which is fatal in up to 83 % of ...

Elsevier2024

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

Digital reconstruction of the mammalian spinal cord: from anatomical reference volume to cell type atlas of the mouse spinal cord

Ihor Kuras

The spinal cord is an elongated nervous structure that together with the brain forms the central nervous system. It relays sensory and motor information between the brain and the body, thus controlling most somatic and autonomic body functions. In recent y ...

EPFL2023

Fast-Spiking Interneurons of the Premotor Cortex Contribute to Initiation and Execution of Spontaneous Actions

Silvestro Micera

Planning and execution of voluntary movement depend on the contribution of distinct classes of neurons in primary motor and premotor areas. However, timing and pattern of activation of GABAergic cells during specific motor behaviors remain only partly unde ...

SOC NEUROSCIENCE2023

Targeted regeneration of spinal cord neurons across a complete injury restores walking

Marco Milano

Spinal cord injury (SCI) interrupts axonal connections between the brain and the spinal cord, and is characterized by a spectrum of sensorimotor and autonomic impairments. While spontaneous recovery is limited, recent studies have shown that functional imp ...

EPFL2023

Comparative Evaluation of Lipofectamine and Dendrimer for Transfection of Short RNA Into Human T47D and MCF-10A Cell Lines

Erfan Shirzadi

Purpose: Non-viral transfection approaches are extensively used in cancer therapy. The future of cancer therapy lies on targeted and efficient drug/gene delivery. The aim of this study was to determine the transfection yields of two commercially available ...

2023

CSF-contacting neurons respond to Streptococcus pneumoniae and promote host survival during central nervous system infection

Ludovic Pierre Gustave Keiser

The pathogenic bacterium Streptococcus pneumoniae (S. pneumoniae) can invade the cerebrospinal fluid (CSF) and cause meningitis with devastating consequences. Whether and how sensory cells in the central nervous system (CNS) become activated during bacteri ...

CELL PRESS2023

Brain matters: unveiling the distinct contributions of region, age, and sex to glia diversity and CNS function

Gioele La Manno, Zahra Moslehi

The myelinated white matter tracts of the central nervous system (CNS) are essential for fast transmission of electrical impulses and are often differentially affected in human neurodegenerative diseases across CNS region, age and sex. We hypothesize that ...

BMC2023

Loss of Motor Cortical Inputs to the Red Nucleus after CNS Disorders in Nonhuman Primates

Simon Borgognon

The premotor (PM) and primary motor (M1) cortical areas broadcast voluntary motor commands through multiple neuronal pathways, including the corticorubral projection that reaches the red nucleus (RN). However, the respective contribution of M1 and PM to co ...

SOC NEUROSCIENCE2023

RNA-based nanomedicines and their clinical applications

Yi Wang

RNA-based nanomedicines encompass a range of therapeutic approaches that utilize RNA molecules or molecules that target RNAs for the treatment or prevention of diseases. These include antisense oligonucleotides (ASOs), small interfering RNAs (siRNAs), endo ...

Tsinghua Univ Press2023

SiliFish: A desktop application to model swimming behavior in developing zebrafish (Danio rerio)

Yann Roussel

SiliFish is an open-source desktop application tomodel and study zebrafish swimming. Here, we explain how to define the general parameters of the model, define cell populations, place them within the spinal cord, and define their projections. We explain ho ...

Elsevier2023

Axonal projections from mouse whisker-related sensory cortex

Yanqi Liu

Neurons primarily communicate through release of neurotransmitter from presynaptic specialisations along their axonal arborisations. In order to understand the functional role of a specific neuron, it is therefore of great interest to know the structure of ...

EPFL2023

Mechanism of KRAS-driven T cell infiltration in colorectal cancer

Amber Dawn Bowler

Colorectal cancer is the second-leading cause of cancer death worldwide. Early-stage disease can be detected with preventative medical screening and is treatable with surgical resection. Sixty-percent of patients, however, are diagnosed with advanced-stage ...

EPFL2023

Soft bioelectronic technologies for selective and multimodal peripheral nerve interfaces

Valentina Marie Paggi

The peripheral nervous system (PNS) regulates the exchange of sensory information and motor commands between the body and the central nervous system. Further, through the autonomic nervous system, the PNS plays a pivotal role in controlling vital physiolog ...

EPFL2023

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

Data-driven whole mouse brain modeling for multi-scale simulations

Dimitri Bruno Marie Rodarie

In this thesis, we present a data-driven iterative pipeline to generate, simulate and validate point-neuron models of the whole mouse brain. The ultimate goal is to replicate close loop experiments with a virtual body in a virtual world. This pipeline was ...

EPFL2022