Aller au contenu principal

Approches génétiques intégrées et nouvelles thérapies pour les maladies rares

Laboratory presentation

INTEGRARE (Integrated Genetic Approaches and New Therapies for Rare Diseases, UMR_S951) is a research unit specializing in translational research and gene therapy of rare genetic diseases. The unit is mainly interested in myopathies, metabolic diseases, blood diseases or immune deficiency. Using recombinant viral vectors (AAV and lentiviral vectors) or CRISPR technologies, researchers design novel therapeutic approaches, defining the pharmacological parameters and the safety of the approaches, and study how to overcome the roadblocks notably to avoid immune responses to gene therapy.
Promising therapeutic candidates are developed towards the clinic through Genethon's therapeutic programs. Several clinical trials of gene therapy are currently underway.
INTEGRARE is a partnership between Généthon, (a private R et D organization created by AFM / Téléthon), Inserm and the Universities (University of Evry / Paris Saclay University, EPHE).

The 20 latest publications

Title Authors Publication date Source
Modern therapeutic approaches to liver-related disorders Antoine Gardin 06/01/22 Journal of Hepatology
Isogenic GAA-KO Murine Muscle Cell Lines Mimicking Severe Pompe Mutations as Preclinical Models for the Screening of Potential Gene Therapy Strategies Valle Montalvo-Romeral, Giuseppe Ronzitti 06/01/22 International Journal of Molecular Sciences
Dual Blockade of Misfolded Alpha-Sarcoglycan Degradation by Bortezomib and Givinostat Combination Lucile Hoch, Celine Bruge, Manon Benabides, Emilie Pellier, Johana Tournois, Gurvan Mahé, Xavier Nissan 04/27/22 Frontiers in Pharmacology
Overcoming the Challenges Imposed by Humoral Immunity to AAV Vectors to Achieve Safe and Efficient Gene Transfer in Seropositive Patients David Alexandre Gross, Novella Tedesco, Christian Leborgne, Giuseppe Ronzitti 04/07/22 Frontiers in Immunology
Co‐Administration of Simvastatin Does Not Potentiate the Benefit of Gene Therapy in the mdx Mouse Model for Duchenne Muscular Dystrophy Nathalie Bourg, Ai Vu Hong, William Lostal, Abbass Jaber, Nicolas Guerchet, Guillaume Tanniou, Fanny Bordier, Emilie Bertil‐froidevaux, Christophe Georger, Nathalie Daniele, Isabelle Richard, David Israeli 02/01/22 International Journal of Molecular Sciences
Durable immunogenicity, adaptation to emerging variants, and low-dose efficacy of an AAV-based COVID-19 vaccine platform in macaques Giuseppe Ronzitti 01/01/22 Molecular Therapy
Lentiviral standards to determine the sensitivity of assays that quantify lentiviral vector copy numbers and genomic insertion sites in cells Guillaume Corre, Ababacar Seye, Sophie Frin, Maxime Ferrand, Céline J. Rocca, Anne Galy 01/01/22 Gene Therapy
Long-term safety and efficacy of lentiviral hematopoietic stem/progenitor cell gene therapy for Wiskott–Aldrich syndrome Anne Galy 01/01/22 Nature Medicine
Cytokines, chemokines and growth factors profile in human aqueous humor in idiopathic uveitis Sylvain Fisson 01/01/22 PLoS ONE
Characterization of Neuromuscular Junctions in Mice by Combined Confocal and Super-Resolution Microscopy Martina Marinello, Caroline Bogni, Jérôme Denard, Ana Buj-Bello 12/08/21 Journal of Visualized Experiments
Hepatic expression of GAA results in enhanced enzyme bioavailability in mice and non-human primates Helena Costa Verdera, Fanny Collaud, Pauline Sellier, Umut Cagin, Julien Fabregue, Simon Barral, Marcelo Simon Sola, Severine Charles, David Alexandre Gross, Pasqualina Colella, Giuseppe Ronzitti, Federico Mingozzi 12/01/21 Nature Communications
Restoring neuronal chloride homeostasis with anti-NKCC1 gene therapy rescues cognitive deficits in a mouse model of Down syndrome Severine Charles, Giuseppe Ronzitti, Federico Mingozzi 10/06/21 Molecular Therapy
Recent Progress in Genome Editing for Gene Therapy Applications: The French Perspective Mario Amendola, Ana Buj-Bello, Marine Laurent, Célia Sourd 10/01/21 Human Gene Therapy
A novel therapeutic strategy for skeletal disorders: Proof of concept of gene therapy for X-linked hypophosphatemia Volha Zhukouskaya, Agnès Linglart, Catherine Chaussain 10/01/21 Science advances
Magnetic resonance imaging is a valuable tool to evaluate the therapeutic efficacy of burosumab in children with X-linked hypophosphatemia Volha Zhukouskaya 10/01/21 European Journal of Endocrinology
Titin M-line insertion sequence 7 is required for proper cardiac function in mice Ariane Biquand, Simone Spinozzi, William Lostal, Isabelle Richard 09/01/21 Journal of Cell Science
Type B coxsackieviruses and central nervous system disorders: critical review of reported associations Sylvain Fisson 07/01/21 Reviews in Medical Virology
Cholesterol metabolism is a potential therapeutic target in Duchenne muscular dystrophy F. Amor, A. Vu Hong, Guillaume Corre, M. Sanson, L. Suel, Isabelle Richard, David Israeli 06/01/21 Journal of Cachexia, Sarcopenia and Muscle
Narrative review of glycogen storage disorder type III with a focus on neuromuscular, cardiac and therapeutic aspects Édouard Berling, Giuseppe Ronzitti 05/01/21 Journal of Inherited Metabolic Disease
Calpains for dummies: What you need to know about the calpain family Simone Spinozzi, Sonia Albini, Heather Best, Isabelle Richard 05/01/21 Biochimica et Biophysica Acta - Proteins and Proteomics

Nombre de publications du laboratoire par domaine scientifique

Chaque publication du laboratoire peut être rangée dans une ou plusieurs disciplines scientifiques : la figure ci-dessus présente le nombre de publications du laboratoire pour chaque discipline de la classification ASJC (Elsevier)