Thérapie génique de la rétine humaine

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Ophtalmologie

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Publications associées (30)

Genetic spectrum of retinal dystrophies in Tunisia

Imen Habibi

We report the molecular basis of the largest Tunisian cohort with inherited retinal dystrophies (IRD) reported to date, identify disease-causing pathogenic variants and describe genotype-phenotype correlations. A subset of 26 families from a cohort of 73 f ...

Nature Research2020

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

Gene Editing Preserves Visual Functions in a Mouse Model of Retinal Degeneration

Diego Ghezzi, Naïg Aurelia Ludmilla Chenais, Paola Vagni, Tommaso Marchetti

Inherited retinal dystrophies (IRDs) are a large and heterogeneous group of degenerative diseases caused by mutations in various genes. Given the favorable anatomical and immunological characteristics of the eye, gene therapy holds great potential for thei ...

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

Single-cell transcriptional logic of cell-fate specification and axon guidance in early-born retinal neurons

Marion Leleu, Gioele La Manno, Pierre Raymond Fabre

Retinal ganglion cells (RGCs), cone photoreceptors (cones), horizontal cells and amacrine cells are the first classes of neurons produced in the retina. However, an important question is how this diversity of cell states is transcriptionally produced. Here ...

2019

Patchy Amphiphilic Dendrimers Bind Adenovirus and Control Its Host Interactions and in Vivo Distribution

Francesco Stellacci, Weina Liu

The surface of proteins is heterogeneous with sophisticated but precise hydrophobic and hydrophilic patches, which is essential for their diverse biological functions. To emulate such distinct surface patterns on macromolecules, we used rigid spherical syn ...

2019

Running Large-Scale Simulations on the Neurorobotics Platform to Understand Vision - The Case of Visual Crowding

Michael Herzog, Gregory Francis, Alban Bornet, Egidio Falotico, Alessandro Ambrosano

Traditionally, human vision research has focused on specific paradigms and proposed models to explain very specific properties of visual perception. However, the complexity and scope of modern psychophysical paradigms undermine the success of this approach ...

2019

Discrete windows of feature integration

Michael Herzog, Adrien Christophe Doerig, Leila Drissi Daoudi - Kleinbauer

A car drives through the night. It is hard to estimate its color from single photoreceptor activity because photoreceptor activation is short and noisy. An efficient way to estimate the color is to average photoreceptor activities along the car’s motion tr ...

SAGE PUBLICATIONS LTD2019

Capacitive-like photovoltaic epiretinal stimulation enhances and narrows the network-mediated activity of retinal ganglion cells by recruiting the lateral inhibitory network

Diego Ghezzi, Naïg Aurelia Ludmilla Chenais, Marta Jole Ildelfonsa Airaghi Leccardi

Photovoltaic retinal prostheses theoretically offer the possibility of a standalone high-resolution electrical stimulation of the retina. However, achieving focused epiretinal stimulation is particularly challenging, because of axonal activation and electr ...

2019

Multilevel regulation of the glass locus during Drosophila eye development

Bart Deplancke, Michael Vincent Frochaux, Abhishek Kumar Mishra

Development of eye tissue is initiated by a conserved set of transcription factors termed retinal determination network (RDN). In the fruit fly Drosophila melanogaster, the zinc-finger transcription factor Glass acts directly downstream of the RDN to contr ...

2019

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