Publication

Allosteric control of type I-A CRISPR-Cas3 complexes and establishment as effective nucleic acid detection and human genome editing tools

Publications associées (36)

Integrated microfluidic tools for the characterization of protein/DNA interactions in vitro and in vivo

The specific interaction between DNA and proteins constitutes one of the crucial elements in the regulation of gene expression. This thesis focuses on the development and optimization of two microfluidic-based technologies called SMiLE-seq and FloChIP that ...

EPFL2019

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

Preservation of visual function in an animal model of retinal degeneration using CRISPR/Cas9 gene editing

Diego Ghezzi, Paola Vagni, Tommaso Marchetti

Aims: Inherited Retinal Disorders represent a difficult target for gene therapy. This study exploits the possibility of using CRISPR/Cas9-based gene editing in photoreceptor progenitors to prevent cell death. We performed all the experiments on Rd10 mice, ...

2018

A selective ER‐phagy exerts procollagen quality control via a Calnexin‐FAM134B complex

Maurizio Molinari

Autophagy is a cytosolic quality control process that recognizessubstrates through receptor-mediated mechanisms. Procollagens,the most abundant gene products in Metazoa, are synthesized inthe endoplasmic reticulum (ER), and a fraction that fails to attaint ...

2018

Engineered anti-CRISPR proteins for optogenetic control of CRISPR-Cas9

Bruno Emanuel Ferreira De Sousa Correia, Zander Harteveld

Anti-CRISPR proteins are powerful tools for CRISPR-Cas9 regulation; the ability to precisely modulate their activity could facilitate spatiotemporally confined genome perturbations and uncover fundamental aspects of CRISPR biology. We engineered optogeneti ...

NATURE PUBLISHING GROUP2018

Gene editing in photoreceptor progenitors prevents visual function loss in a mouse model of retinal degeneration

Diego Ghezzi, Paola Vagni

Purpose:Currently, there is no known cure for Retinitis pigmentosa (RP). Even if some treatments can slow down the progression of the disease, none of them can effectively stop retinal degeneration. We exploited the possibility of an early intervention in ...

2017

Cell lineage tracing in human tissues using somatic mitochondrial mutations

Paul Refinetti

Lineage tracing is a basic concept in developmental biology, as well as diseases related to organ development. A reliable method to track cell lineages in human tissues has the potential to greatly contribute to the fields of oncogenesis, tissue renewal, a ...

EPFL2017

Preventing visual function loss in the rd10 mouse model of retinitis pigmentosa using gene editing

Diego Ghezzi, Paola Vagni

Currently, there is no known cure for retinitis pigmentosa (RP). Even if some treatments can slow down the progression of the disease, none of them can effectively stop retinal degeneration. This study exploits the possibility of an early intervention in p ...

2017

Mapping mitochondrial heteroplasmy in a Leydig tumor by laser capture microdissection and cycling temperature capillary electrophoresis

Stephan Morgenthaler, Paul Refinetti, Per Olaf Ekstrom

Background: The growth of tumor cells is accompanied by mutations in nuclear and mitochondrial genomes creating marked genetic heterogeneity. Tumors also contain non-tumor cells of various origins. An observed somatic mitochondrial mutation would have occu ...

BioMed Central2017

Nanocapillaries combined with optical tweezers as a single molecule technique for studying DNA-protein complexes

Roman Bulushev

Interactions of proteins with DNA are essential for carrying out DNA's biological functions and performing a cellular cycle. Such processes as DNA replication, expression and repair are performed by an organised action of various proteins. To better unders ...

EPFL2017

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