Publications related to Adeno-associated virus

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

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

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

The role of medial amygdala inhibitory neurons in regulating social behavior

Aiste Baleisyte

Aggression is an evolutionary conserved social behavior that is necessary for an animalÂŽs survival. The neural substrates coordinating aggression are known to exist in the amygdala and the hypothalamus. While the medial amygdala (MeA) and the ventrolatera ...

EPFL2021

Gene replacement therapy provides benefit in an adult mouse model of Leigh syndrome

Johan Auwerx

Mutations in nuclear-encoded mitochondrial genes are responsible for a broad spectrum of disorders among which Leigh syndrome is the most common in infancy. No effective therapies are available for this severe disease mainly because of the limited capabili ...

2020

Efficient viral transduction in mouse inner ear hair cells with utricle injection and AAV9-PHP.B

Bernard Schneider, Paola Andrea Solanes Vega

Viral delivery of exogenous coding sequences into the inner ear has the potential for therapeutic benefit for patients suffering genetic or acquired hearing loss. To devise improved strategies for viral delivery, we investigated two injection techniques, r ...

2020

Dopaminergic neurodegeneration induced by Parkinson's disease-linked G2019S LRRK2 is dependent on kinase and GTPase activity

Elpida Tsika, Darren Moore, Xi Chen

Mutations in leucine-rich repeat kinase 2 (LRRK2) are the most common cause of late-onset, autosomal-dominant familial Parkinson's disease (PD). LRRK2 functions as both a kinase and GTPase, and PD-linked mutations are known to influence both enzymatic acti ...

National Academy of Sciences2020

Scalable Production of AAV Vectors in Orbitally Shaken HEK293 Cells

Florian Maria Wurm, Bernard Schneider, Vivianne Padrun, Nicole Déglon, Daniel Roman Blessing, Gabriel Vachey

Adeno-associated virus (AAV) vectors are currently among the most commonly applied for in vivo gene therapy approaches. The evaluation of vectors during clinical development requires the production of considerable amounts of highly pure and potent vectors. ...

2019

Delivery of CRISPR/Cas9 using AAV-PHP.B in the inner ear leads to allele-specific inactivation of the mutated Tmc1 allele and protects auditory function in Beethoven mice

Bernard Schneider, Paola Andrea Solanes Vega, Sofia Carmen Suzanne Spataro

Hearing Loss (HL) is the most prevalent sensorineural disorder, affecting 360 million people worldwide. As genetic causes lead to 50% of pre-lingual deafness, gene therapy is considered as a potential therapeutic strategy. Mutations in the TMC1 gene are li ...

MARY ANN LIEBERT, INC2019

Optogenetic Peripheral Nerve Immunogenicity

Optogenetic technologies have been the subject of great excitement within the scientific community for their ability to demystify complex neurophysiological pathways in the central (CNS) and peripheral nervous systems (PNS). The excitement surrounding opto ...

Nature Research2018

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

Assessment of brain metabolite correlates of AAV-mediated overexpression of human alpha-synuclein in cortical neurons by in vivo(1) H-MR spectroscopy at 9.4 T

Sarah Catherine Sonnay

In this study, we used proton-localized spectroscopy ((1) H-MRS) for the acquisition of the neurochemical profile longitudinally in a novel rat model of human wild type alpha-synuclein (a-syn) overexpression. Our goal was to find out if the increased a-syn ...

Wiley-Blackwell2016

Lack of additive role of ageing in nigrostriatal neurodegeneration triggered by alpha-synuclein overexpression

Grégoire Courtine, Laetitia Danielle Philippine Baud, Isabel Laura Vollenweider

Introduction: Parkinson's disease (PD) is a progressive neurodegenerative disorder characterized by the loss of dopaminergic neurons as well as the presence of proteinaceous inclusions named Lewy bodies. alpha synuclein (alpha-syn) is a major constituent o ...

2015

Study of transposon-mediated cell pool and cell line generation in CHO cells

Sowmya Balasubramanian

The goal of this thesis was to evaluate the use of artificial transposon systems for the generation of recombinant cell pools and cell lines with Chinese hamster ovary (CHO) cells as the production host. Transposons are naturally occurring genetic elements ...

EPFL2015

Intracerebroventricular Injection of Adeno-Associated Virus 6 and 9 Vectors for Cell Type-Specific Transgene Expression in the Spinal Cord

Patrick Aebischer, Bernard Schneider, Elisabeth Anne Dirren, Christopher Towne, Veronica Setola

In the context of motoneuron diseases, gene delivery as an experimental or therapeutic approach is hindered by the challenge to specifically target cell populations that are widely distributed along the spinal cord. Further complicating the task, transgene ...

Mary Ann Liebert2014

Direct and Retrograde Transduction of Nigral Neurons with AAV6, 8, and 9 and Intraneuronal Persistence of Viral Particles

Patrick Aebischer, Bernard Schneider

Recombinant adeno-associated viral (AAV) vectors of serotypes 6, 8, and 9 were characterized as tools for gene delivery to dopaminergic neurons in the substantia nigra for future gene therapeutic applications in Parkinson's disease. While vectors of all th ...

Mary Ann Liebert2013

Peptide-Based Vaccinology: Experimental and Computational Approaches to Target Hypervariable Viruses through the Fine Characterization of Protective Epitopes Recognized by Monoclonal Antibodies and the Identification of T-Cell-Activating Peptides

Matteo Dal Peraro, Matteo Castelli

Defining immunogenic domains of viral proteins capable of eliciting a protective immune response is crucial in the development of novel epitope-based prophylactic strategies. This is particularly important for the selective targeting of conserved regions s ...

Hindawi Publishing Corporation2013

MAR Elements and Transposons for Improved Transgene Integration and Expression

Nicolas Mermod, Déborah Ley, Niamh Harraghy

Reliable and long-term expression of transgenes remain significant challenges for gene therapy and biotechnology applications, especially when antibiotic selection procedures are not applicable. In this context, transposons represent attractive gene transf ...

2013

Subtle differences in virus composition affect disinfection kinetics and mechanisms

Tamar Kohn, Brian Pecson, Krista Rule Wigginton, Anne Thérèse Sigstam, Michele Cascella

Viral disinfection kinetics have been studied in depth, but the molecular-level inactivation mechanisms are not understood. Consequently, it is difficult to predict the disinfection behavior of non-culturable viruses, even when related, culturable viruses ...

American Society for Microbiology2013

Study of the improved Sf9 transient gene expression process

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

Insect cells have been widely used for the production of recombinant proteins using recombinant baculovirus for gene delivery [1]. To simplify protein production in insect cells, we have previously described a method, based on transient gene expression (TG ...

2013

Adeno-Associated Virus Activates an Innate Immune Response in Normal Human Cells but Not in Osteosarcoma Cells

Peter Martin Beard

Adeno-associated virus (AAV) is a small, DNA-containing dependovirus with promising potential as a gene delivery vehicle. Given the variety of applications of AAV-based vectors in the treatment of genetic disorders, numerous studies have focused on the imm ...

American Society for Microbiology2011