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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

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Publications associées (33)

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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

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

Human Cochlear Nucleus on 7 Tesla Diffusion Tensor Imaging: Insights Into Micro-anatomy and Function for Auditory Brainstem Implant Surgery

Nicolas Vachicouras

Objective:The cochlear nucleus (CN) is the target of the auditory brainstem implant (ABI). Most ABI candidates have Neurofibromatosis Type 2 (NF2) and distorted brainstem anatomy from bilateral vestibular schwannomas. The CN is difficult to characterize as ...

LIPPINCOTT WILLIAMS & WILKINS2020

Comparison of Responses to DCN vs. VCN Stimulation in a Mouse Model of the Auditory Brainstem Implant (ABI)

Stéphanie Lacour, Nicolas Vachicouras, Florent-Valéry Coen

The auditory brainstem implant (ABI) is an auditory neuroprosthesis that provides hearing to deaf patients by electrically stimulating the cochlear nucleus (CN) of the brainstem. Whether such stimulation activates one or the other of the CN's two major sub ...

SPRINGER2022

Assessing Public Opinion on CRISPR-Cas9: Combining Crowdsourcing and Deep Learning

Marcel Salathé, Martin Mathias Müller

Background: The discovery of the CRISPR-Cas9-based gene editing method has opened unprecedented new potential for biological and medical engineering, sparking a growing public debate on both the potential and dangers of CRISPR applications. Given the speed ...

JMIR PUBLICATIONS, INC2020

Computational design of anti-CRISPR proteins with improved inhibition potency

Wei Sun, Bruno Emanuel Ferreira De Sousa Correia, Sandrine Madeleine Suzanne Georgeon, Stéphane Rosset, Zander Harteveld, Andreas Scheck

Anti-CRISPR (Acr) proteins are powerful tools to control CRISPR-Cas technologies. However, the available Acr repertoire is limited to naturally occurring variants. Here, we applied structure-based design on AcrIIC1, a broad-spectrum CRISPR-Cas9 inhibitor, ...

NATURE PUBLISHING GROUP2020

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

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

Johan Auwerx, Giorgia Benegiamo

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

Prenatal auditory experience and its sequelae

Lukas Vogelsang

Towards the end of the second trimester of gestation, a human fetus is able to register environmental sounds. This in utero auditory experience is characterized by comprising strongly low-pass-filtered versions of sounds from the external world. Here, we p ...

2022

An optimized high throughput platform, from genetic transformation to fermentation, to screen for secreted laccases in Aspergillus niger

Laure Marie Colette Leynaud-Kieffer Curran

Many challenges are faced in the conversion of biomass into advanced biofuels, one of which is finding the correct organism for the job. The filamentous fungus Aspergillus niger has been chosen as a biocatalyst for cellulose, hemicellulose, and lignin degr ...

EPFL2021

In vivo evaluation of gene editing and prosthetic strategies to restore vision in small and large animal models of retinal degeneration

Paola Vagni

Inherited retinal diseases (IRDs) form a group of diverse disorders that lead to the degeneration of the light-sensing cells of the retina: the photoreceptors. IRDs are among the leading causes of blindness in working-age adults living in industrialized co ...

EPFL2020

Selection of Water-Soluble Chitosan by Microwave-Assisted Degradation and pH-Controlled Precipitation

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

In the field of gene therapy, chitosan (CS) gained interest for its promise as a non-viral DNA vector. However, commercial sources of CS lack precise characterization and do not generally reach sufficient solubility in aqueous media for in vitro and in viv ...

2020

Human Head Yaw Estimation based on Two 3-axis Accelerometers

Hervé Lissek, Gilles André Courtois, Vincent Pierre Olivier Grimaldi

The estimation of the orientation of an object, and a human head in particular, can be defined by the Euler angles: the yaw, pitch and roll. The robust and drift-free estimation of those angles is usually achieved with the data from several sensors such as ...

2022