Concept

Édition génomique

Publications associées (110)

A computationally designed chimeric antigen receptor provides a small-molecule safety switch for T-cell therapy

Bruno Emanuel Ferreira De Sousa Correia, George Coukos, Pablo Gainza Cirauqui, Jaume Bonet Martinez, Sabrina Susan Vollers, Sailan Shui

The activity of CAR-T cells is reversibly halted with a small-molecule drug. Approaches to increase the activity of chimeric antigen receptor (CAR)-T cells against solid tumors may also increase the risk of toxicity and other side effects. To improve the s ...

2020

A new approach to Cas9-based genome editing in Aspergillus niger that is precise, efficient and selectable

Aspergillus niger and other filamentous fungi are widely used in industry, but efficient genetic engineering of these hosts remains nascent. For example, while molecular genetic tools have been developed, including CRISPR/Cas9, facile genome engineering of ...

2019

La modularité au service des espaces d'enseignement

Répondre aux besoins d'espaces scolaires confronte les responsables municipaux à des questions touchant simultanément à l'espace et au temps. Au sein d'un territoire donné, il n'est ainsi pas rare que des décalages apparaissent entre les effectifs scolaire ...

2019

Allele-specific gene editing prevents deafness in a model of dominant progressive hearing loss

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

Since most dominant human mutations are single nucleotide substitutions(1,2), we explored gene editing strategies to disrupt dominant mutations efficiently and selectively without affecting wild-type alleles. However, single nucleotide discrimination can b ...

NATURE PUBLISHING GROUP2019

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

Diego Ghezzi, Paola Vagni, Naïg Aurelia Ludmilla Chenais, 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

A ligand-based system for receptor-specific delivery of proteins

Kai Johnsson, Luc Reymond

Gene delivery using vector or viral-based methods is often limited by technical and safety barriers. A promising alternative that circumvents these shortcomings is the direct delivery of proteins into cells. Here we introduce a non-viral, ligand-mediated p ...

Springer2019

Gene drives for schistosomiasis transmission control

Francisco Javier Perez Saez

Schistosomiasis is one of the most important and widespread neglected tropical diseases (NTD), with over 200 million people infected in more than 70 countries; the disease has nearly 800 million people at risk in endemic areas. Although mass drug administr ...

2019

Hominoid-Specific Transposable Elements and KZFPs Facilitate Human Embryonic Genome Activation and Control Transcription in Naive Human ESCs

Didier Trono, Priscilla Turelli, Evaristo Jose Planet Letschert, Julien Léonard Duc, Sandra Eloise Kjeldsen, Alexandre Coudray, Julien Paul André Pontis, Julien Duc

Expansion of transposable elements (TEs) coincides with evolutionary shifts in gene expression. TEs frequently harbor binding sites for transcriptional regulators, thus enabling coordinated genome-wide activation of species- and context-specific gene expre ...

2019

Enhanced flux prediction by integrating relative expression and relative metabolite abundance into thermodynamically consistent metabolic models

Vassily Hatzimanikatis, Noushin Hadadi, Vikash Kumar Pandey

The ever-increasing availability of transcriptomic and metabolomic data can be used to deeply analyze and make ever-expanding predictions about biological processes, as changes in the reaction fluxes through genome-wide pathways can now be tracked. Current ...

2019

Phosphorylation of the VAR2CSA extracellular region is associated with enhanced adhesive properties to the placental receptor CSA

Romain Hamelin, Florence Armand, Marc Moniatte

Plasmodium falciparum is the main cause of disease and death from malaria. P. falciparum virulence resides in the ability of infected erythrocytes (IEs) to sequester in various tissues through the interaction between members of the polymorphic P. falciparu ...

PUBLIC LIBRARY SCIENCE2019

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