Wednesday April 11th, 2018: “Gene Therapy for Sickle Cell Disease” Pr Donald B. Kohn , Professor of Microbiology, Immunology and Molecular Genetics (MIMG); Pediatrics; and Molecular & Medical Pharmacology, Director of the UCLA Human Gene and Cell Therapy Program, Kohn Stem Cell Gene Therapy Lab, Depts. of M.I.M.G., Pediatrics and MMP, University of California, Los … [Read more]
Wednesday March 21th, 2018: «Recombinant AAV for Human Gene Therapy: Progress, Challenges and Perspectives for Vector Design, Manufacturing and Characterization» Pr Fraser Wright, Co-Founder and Chief Technology Advisor / Officer at Spark Therapeutics, former Scientific Director, Clinical Vector Core at the University of Pennsylvania and former Director of Development and Manufacturing at Avigen.15
Tuesday March 13th, 2018 : « Signaling Properties of Stem Cells and their Cargo Vesicles » Dr. Stefano Pluchino, University Reader in Regenerative Neuroimmunology and Honorary Consultant in Neurology, Department of Clinical Neurosciences, University of Cambridge, United Kingdom.
Le mardi 12 septembre 2017 : “Single domain antibodies as potential therapeutic tools in bleeding disorders” Dr. Peter Lenting, Director of Research , Inserm U1176 , Haemostasis-Inflammation-Thrombosis, CHU Bicêtre, Kremlin Bicêtre, Paris, France.
Tuesday June 27th 2017 : “Patient-specific iPSCs for disease modelling and cell therapy applications in lysosomal storage diseases” Dr. Angela Gritti, Head of Gene/neural stem cell therapy for lysosomal storage diseases Unit at the San Raffaele Telethon Institute for Gene Therapy, Milan, Italy.10
Monday June 19th,2017: “MS based tools for bioprocess optimization and product characterization” Dr. Patricia Alves, Senior scientist, Health & Pharma Division, Animal Cell Technology Unit, Cell Bioprocesses Laboratory, iBET, Portugal.
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.