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- Questions and answers about gene therapy
- What is gene therapy ?
- Gene therapy: How it works?
- Are there different approaches in gene therapy?
- Which diseases are treated by gene therapy?
- Are there risks associated with gene therapy?
- What are the main steps before market access of a drug derived from gene therapy?
- Is there already on the market gene therapy drugs? For which indications?
- What is the stage of the current research ?
- What are the ethical issues associated with gene therapy?
- Gene therapy: a treatment for rich people?
- Educational Resources
- Questions and answers about gene therapy
- Contact us
Posts by category
- Category: News
- Sanofi, WhiteLab Genomics, the TaRGeT Laboratory at Nantes University, and
Institut Imagine launch WIDGeT to make France a global leader in gene therapy - Annarita Miccio receives 1st prize of the Paris Region Innovators Award 2023 for her work on hemoglobinopathies
- Accelerating gene therapy: assessment and prospects at the end of the DIM Gene Therapy program
- First in vivo success of a gene therapy for the maple syrup urine disease
- A new tool to correct a mutation in beta-thalassemia
- “The systematic screening of sickle cell disease extended to the entire French population is a real victory”
- First successes and new approaches in gene therapy
- Innovators Award 2021: Deniz Dalkara laureate for her results on visual restoration by gene therapy
- Quel est le rôle des associations de patients au sein du DIM Thérapie Génique ?
- Webinars SFTCG
- CRISPR-Cas9, molecular scissors at the service of the gene therapies of tomorrow : interview from Dr. Annarita Miccio, lab director at the Imagine Institute, Paris
- Gene therapy combines cell therapy to treat blindness: interview from Dr Deniz Dalkara, tenured researcher at Institut de la Vision, Paris
- A gene therapy trial treats 6 patients with chronic granulomatous, a rare immune disorder
- Sanofi, WhiteLab Genomics, the TaRGeT Laboratory at Nantes University, and
- Category: Anounce
- Back to the GTRU 2022, the closing event of the DIM Thérapie Génique
- Crispr-Cas9 molecular scissors used for the first time in human to treat inherited blindness
- Two CAR T drugs, awarded the Prix Galien France 2019 in the “Innovative therapy medicine” category
- France Culture dédie une de ces émissions à la thérapie génique
- Gensight LUMEVOQ™, a gene therapy treatment will be used in Paris to treat blindness
- First CRISPR treatment shows promising results for 2 patients suffering from blood disorder
- Le DIM Thérapie Génique sensibilise le grand public à la thérapie génique
- Journées Européennes du Patrimoine 2019
- A major research project in gene therapy is financed thanks to the RHU call of projects
- Dr. Annarita Miccio team obtains an AFM Telethon grant for the sickle cell diseases project
- FDA approved a new gene therapy treatment in Spinal Muscular Atrophy
- Interview: Nick Leschly, CEO at Bluebird Bio
- New approval in gene therapy for the treatment of beta-thalassemia
- The hearing restored by gene therapy in the mouse model
- 2018 ASH Award for a DIM project
- Back to the GTRD – Feb 7 & 8th at Imagine Institute
- Back to the Gene Therapy Partnering Day – September 17th at Imagine Institute
- Category: Job offer
- JOB OFFER – 18 months postdoctoral position in Imagine Institute: AAV gene therapy for Maple Syrup Urine Disease (MSUD)
- OFFRE DE STAGE – Master 1 Recherche : Impact of dystrophin restoration on neurological disorders in a mouse model
- OFFRE D’EMPLOI – BAC+3 -ASSISTANT INGENIEUR (H/F)
- OFFRE D’EMPLOI – Thèse : Etude de la vasculopathie cérébrale drépanocytaire
- OFFRE DE STAGE – BAC+2 – PLATEFORME DE NEURO-COMPORTEMENT
- JOB OFFER – ENGINEER POSITION (M/F) 16 MONTHS
- OFFRE DE STAGE LICENCE – Validation de vecteurs adéno-associés (AAV) pour des études immunologiques
- Offre de stage Master 2 – Etude la vasculopathie cérébrale chez les enfants drépanocytaires dans un modèle carotidien personnalisé imprimé en 3D
- OFFRE DE STAGE MASTER 2 – Optimisation de l’analyse globale du plasma par protéomique à très haute débit
- OFFRE DE STAGE MASTER – Gene editing in Sickle Cell Disease
- OFFRE D’EMPLOI – TECHNICIEN EN RECHERCHE & DEVELOPPEMENT H/F (CDD 12 mois)
- OFFRE DE STAGE LICENCE– Production de progéniteurs lymphoïdes génétiquement modifiés
- OFFRE DE STAGE LICENCE – Vecteurs de thérapie génique
- OFFRE DE STAGE BTS/Licence – Développement des activités en phospho-protéomique de la plateforme 3P5 de l’Institut Cochin
- OFFRE DE STAGE – Analyse par cytométrie en flux et tri cellulaire de la différenciation érythroïde humaine
- OFFRE DE STAGE – Technicien de laboratoire en Recherche et Développement
- OFFRE DE STAGE MASTER 2 – Mise en place d’un essai de thérapie génique préclinique pour l’ataxie de Friedreich
- OFFRE DE STAGE Master 2 – Mesure du fonctionnement cérébral au repos dans les troubles du spectre de l’autisme par la méthode d’IRM-ASL
- OFFRE DE STAGE Master 2 – Optimisation de l’expansion en condition de grade clinique de lymphocytes T régulateurs humains pour la transplantation d’organes solides
- OFFRE DE STAGE – Titration de vecteur adéno-asssociés (AAV) par digitale droplet PCR
- Offre de stage – Technicien en recherche médicale (H/F)
- Assistant Engineer position CDD 12 month
- Category: Results
- Category: Call for proposals
- Appel à projet 2022 du DIM Thérapie Génique – Vague 3
- Vague 2 de l’appel à projet DIM Thérapie Génique 2021
- Paris Region Fellowship programme 2021: applications for Marie-Curie post-doc fellowships are now open!
- Call for Proposals DIM Gene Therapy 2021
- Prolongation jusqu’au 25 mars de l’appel à candidatures Paris Region PhD 2021 en thérapie génique
- Paris Region PhD 2021 : appel à candidatures pour le financement d’allocations doctorales en thérapie génique
- Paris Region launches in 2020 its first Marie-Curie call of proposal for 26 post-doctoral positions. Applications are open!
- Call for Proposal DIM Thérapie Génique 2020
- Paris Region PhD 2020: appel à candidatures pour le financement d’allocations doctorales en thérapie génique
- Call for proposal DIM Gene Therapy 2019
- Exceptionnal extension – Call for Proposal DIM 2019
- Call for Proposal DIM Thérapie Génique 2019
- Call for proposal for PhD fellowship
- Call for Projects DIM Thérapie Génique 2018
- Call for Projects DIM Thérapie Génique 2017
Events
- Symposium 2022 - The Gene Therapy Revolution Underway: The Latest Advances
- "Gene therapy, between hopes and realities" conference - February 28 2022
- 53e annual meeting of the French Society for Immunology – postponed to December 7-9, 2021
- Cell Therapy manufacturing and Gene Therapy congress
- Annual meeting of SFTCG
- Quand l'ADN devient médicament
- Journées Européennes du Patrimoine 2019
- CRISPR/Cas9 seminar at Imagine Institute
- Gene Therapy for Rare monogenic Diseases Symposium 2019
- Gene Therapy Partnering Day 2018
Supported projects
- N202001 - Circulating cell-free SMN RNAs as therapeutic monitoring biomarker in spinal muscular atrophy
- M202001 - Gene therapy of mitochondrial diseases
- T202001 - Donor-specific regulatory T cell therapy in transplantation through CAR engineering
- D202001 - Optimizing a Genome-Editing Protocol to treat Sickle Cell Disease patients
- D202002 - Sickle cell disease-related cerebral vasculopathy study
- N202002 - Sensitive Fluorescence scanner as an accelerator of gene therapy assessment
- V202001 - Gene and cell therapy for visual restoration
- N202003 - Purchase of a Transmission Electron Microscope with a cryogenic object holder and of a Cryoplunger
- AA202001 - Gene therapy for X-linked hypophosphatemia
- N202004 - Therapeutic approaches by modulating mRNA splicing for the treatment of neuromuscular diseases
- M202002 - High sensitivity metabolomics for MSUD gene therapy
- D202003 - Preclinical Studies for Genome-Editing based-Treatment of Sickle Cell Disease
- D202004 - Effect of hypoxia on erythroid differentiation in sickle cell disease
- D202005 - Automated learning for characterization in flow imaging of markers of therapeutic efficacy in patients with sickle cell disease treated by gene therapy
- D202006 - Measurement of the hemoglobin mass of sickle cell patients treated by gene therapy using the "CO rebreathing" method
- MA202001 - Proteomic analysis to support gene therapy projects
- MA202002 - Diagnosis and monitoring of pre and post gene therapy patients at the Biological Resource Center (CRB-DNA) of Necker Hospital
- T202002 - Optimizing Hematopoietic Stem and Progenitor Cells (HSPC) genetic engineering in inflammatory context
- T202003 - Pre-clinical model for the use of universal MAIT cells carrying a chimeric receptor (CAR-MAIT)
- D201906 - Cellular characterization of the red blood cells deformability of sickle cell patients treated with gene therapy
- T201905 - Use of universal MAIT cells carrying a chimeric receptor (CAR-MAIT) in an allogenic context
- H201904 – Deserythrocytation of bone marrow in gene therapy protocols
- A201901- Pushing forward cochlear gene therapy with a high speed and large field of view confocal microscope
- D201905 - Intravascular hemolysis assessment in Sickle Cell Patients treated with Gene Therapy
- I201901 - Multimodal brain imaging in patients treated with gene therapy
- H201903 - HIV treatment with genetically modified T progenitors resistant to infection
- N201902 - Quantitative measurement of antisense oligonucleotide effects on SMN transcript isoforms in spinal muscular atrophy
- D201904 - Platform for the study of differentiation and cell dynamics during normal and pathological erythropoiesis
- H201902 - Development of new gene therapy lentiviral vectors for HIV patients
- T201904 - Assessment of inflammatory cytokines in gene therapy protocols
- D201903 - Functional validation by microfluidics of the gene therapy effect on sickle red blood cells
- D201902 - Proteomic analysis of blood cells of sickle cell patients before and after gene therapy
- V201901 - CRX gene therapy in retinal dystrophies mouse models : proof of concept for CRX dominant mutations as well as CRX independent retinopathies
- N201901 - Live cell videomicroscopy of gene therapy tools in culture models of disease
- T201903 - Tolerance by genetic engineering of regulatory T cells
- H201901 - HIV vaccine approaches by genetic engineering of B-cells
- T201902 - Quality control of advanced therapy products and biological monitoring of patients post gene therapies
- T201901 - Monitoring of the gene therapy efficiency and study of the immunological reconstitution of patients with immune deficits by determining the copy number into the cells
- M201901 - Quantification of the mitochondrial and dermatological gene therapies effects by 3D live cell videomicroscopy
- D201901 - Development of an in vitro model for cerebral vasculopathy monitoring in gene therapy of sickle cell disease
- T201804 -T-reg-mediated tolerance induction with AAV vectors
- T201803 - Regulating gene therapy by recruitment of autoimmune antibodies
- T201802 - Transversal Cell Therapy Treg / CAR-Treg
- V201801 - Treatment of corneal stem cell deficiencies by gene and cell therapy
- AA201801 - Development of a platform for the production of recombinant AAV vectors
- M201801 - Gene therapy in Friedreich’s Ataxia
- H201801 - Inversed motorized microscope equipped for infected live-cell imaging
- T201801 - Curative treatment of severe haemophilia A
- V201802 - Gene therapy independent of mutations for retinal dystrophies
- D201801 - Study of erythropoiesis and circulating red blood cells in gene therapy for sickle cell disease
- D201701- Development of new therapeutic strategies in sickle cell disease
- D201702 - Preclinical development of new lentiviral vectors and gene therapy strategies to treat sickle cell disease
- D201703 - New gene therapy strategy for sickle cell disease using umbilical cord blood
- M201701 - Gene therapy of mitochondrial diseases
- M201702 - Gene therapy of Maple Sirup Urine Disease (MSUD)
- A201701 - Gene therapy of auditory damage
- AA201701 - Development of AAV vector platform for R&D purposes
- O201701 - Establishment of proof of concept for the gene therapy-modified bone marrow transplant test in osteoconductive and osteolytic constitutional bone pathologies: study of cellular models
- D201704 - Acquisition of a new generation sequencer
- I201701 - Development of a small animal imaging platform
- N201701 - Quantitative approach of antisense oligonucleotides effect on targeted RNA in Spinal muscular atrophies therapeutic trials
Réseau
- Institut de Myologie
- Université Paris Cité
- Institut de la Vision
- CNRS
- Région île-de- France
- Medicen
- Institut de l’Audition
- Hôpital Necker
- Hôpital Robert Debré
- Hôpital Henri Mondor
- Hôpital Kremlin Bicêtre
- Genethon
- EFS
- APHP
- Inserm
- L'Institut Imagine
Annuaire / Membres
- Marina Cavazzana
- Jean-Daniel Lelièvre
- Corinne Antignac
- Christine Bodemer
- Alain Hovnanian
- Vincent Mouly
- Agnès Rötig
- Olivier Goureau
- Deniz Dalkara
- Fulvio Mavilio
- Federico Mingozzi
- Julien Zuber
- Judith Melki
- Pascale de Lonlay
- Matthias Titeux
- Nathalie Boddaert
- Christine Petit
- Pablo Bartolucci
Catégories Projets soutenus
Sessions AAP
Maladies
- AIDS
- autoimmune diseases
- blindness
- Friedreich's Ataxia
- Ghosal syndrome
- haemophilia A
- hereditary deafness
- Leigh syndrome associated with LRPPRC gene mutations
- MSUD
- Sickle cell disease
- Spinal muscular atrophies (SMA)
- Usher syndrome
Axe de recherche
- Audition
- Sickle cell disease
- Imagery
- Metabolic and mitochondrial diseases
- Neurodegenerative diseases
- Skin
- Kidney
- Tolerance and Immunity
- Viral vectors
- HIV
- Vision
Catégorie de membres
- HIV research coordinator
- Kidney research coordinator
- DIM Thérapie Génique coordinator
- Skin research coordinator
- Imaging research coordinator
- Viral vectors research coordinator
- Tolerance and Immunity research coordinator
- Neurodegenerative diseases research coordinator
- Metabolic and Mitochondrial diseases research coordinator
- Audition research coordinator
- Sickle cell disease research coordinator
- Vision research coordinator
- Research coordinator
