The teams of Genethon and the Institut de Myologie –the AFM-Telethon laboratories-, and a team from the University of Ferrara, announce in Molecular Therapy Nucleic Acids that they have succeeded in restoring the expression of dystrophin, thanks to CRISPR-Cas9! Correction of the Exon 2 Duplication in DMD Myoblasts by a Single CRISPR/Cas9 System Exonic duplications account … [Read more]
The AFM-Telethon announced on Monday 20th March : The 30th Telethon edition reaches a final collection of 92,740,769 euros ! On 2nd and 3rd December 2016, the Telethon enthralled the public throughout France. The final collection figure now allows us to pursue the objective we wet ourselves 30 years ago: to find a cure! Thank … [Read more]
Tuesday February 28th , 2017: “Tryptophan metabolism control inhibitor development in hemophilia A” Dr. Francesca Fallarino, Associate Prof. at the University of Perugia Department of Experimental Medicine, University of Perugia, in Perugia, Italy.
Wednesday February 22d, 2017: “Research for rare disease: RD-Connect and the importance of data sharing” Pr. Hanns Lochmüller, MD, FAAN, Chair of Experimental Myology, Deputy Director, Institute of Genetic Medicine, The John Walton Muscular Dystrophy Research Centre – MRC Centre for Neuromuscular Diseases. Newcastle University , Newcastle upon Tyne- United Kingdom.
Monday January 23rd, 2017: “Rescue of exon skipping defects using modified U1 snRNAs: a novel therapeutic approach for spinal muscular atrophy” Prof. Franco Pagani, Head of Human Molecular Genetics, International Centre for Genetic Engineering and Biotechnology(ICGEB), Trieste, Italy.
Wednesday 18th January, 2017: “From viruses to designer nanoparticles – Tailoring adeno-associated viruses for gene therapy” Prof. Dr. Hildegard Büning, Head of the Laboratory for Infection Biology & Gene Transfer in the Institute of Experimental Hematology at Hannover Medical School, Hannover, Germany.
Friday 18th November, 2016: “Gene Editing to Correct Chronic Granulomatous Disease” Prof. Harry Malech, Chief, Laboratory of Host Defenses; Head, Genetic Immunotherapy Section; National Institute of Allergy and Infectious Diseases; National Institutes of Health. Bethesda, MD, USA.
Tuesday 4th October, 2016: “Targeting FVIII expression in endothelial and myeloid cells for hemophilia a gene therapy in mice” Prof. Antonia Follenzi, Associate professor of Histology, Università degli Studi del Piemonte Orientale “Amedeo Avogadro”, Dipartimento di Scienze della Salute (Dept. of Health Sciences) in Novara, Italy.
Tuesday 21th June,2016: “Du génome au métabolite” Marcel Salanoubat, Directeur de l’UMR 8030, « Génomique Métabolique », Genoscope, Evry, France.
Tuesday 14th June 2016: “Gene therapy for liver genetic diseases” Dr Gloria González Aseguinolaza, Director of the Gene Therapy and Regulation of Gene Expression program, Head of the laboratory of Gene therapy for liver diseases, CIMA, University of Navarra, Pamplona, Spain.