Genethon

📢 [#ESGCT2024] 💥A wonderful week at #ESGCT2024 for Genethon delegates with 18 posters and 4 oral presentations. Thank you to all the researchers from different teams for their participation, for the impressive scientific presentations, sharing their latest advances and results in #GeneTherapy. Save the date and see you on #ESGCT2025, 7-10 October in Sevilla!   👏Louisa Jauze Braun Serge Edith Renaud-Gabardos Maëlle RALU Valentina Buffa Christian LEBORGNE Sonia Albini, PhD  Maxime Ferrand Abbass J. Ai Vu Hong Novella Tedesco Ricardo Rojas Louise Mangin Sophie Olivier Emmanuel Thévenot Marion DEROME Odile Boespflug Tanguy Amandine FRANÇOIS Jérôme DENARD ALEXANDRA TACHTSIDI Martina Marinello Anne Galy Frederic Revah Mario Amendola Giuseppe Ronzitti Gérald Perret (Ph.D, MBA) Céline Contet Claire Bichat 

📢 [ESGCT congress] 👏Thank you Maëlle RALU PhD student Genethon for presenting your work about using of a CRISPR-Cas 9 strategy, in the context of DMD in mice, to over-express utrophin in cells, a protein that can compensate for the absence of dystrophin, and thus improve the phenotype. “Our next goal is to improve the system using different AAV capsids, but also to evaluate the functional benefits of the treatment in mice” explains Maëlle RALU.   #genethon #congress #ESGCT2024 #presentation #gene editing #DMD Mario Amendola European Society of Gene and Cell Therapy

📢 [ESGCT congress] 🫵SAVE THE DATE: Today from 06:00 pm - 07:30 pm CEST time - Come and ask your questions to Anne Galy with her poster: Reduction of muscle fibrosis by FAP-CAR T cells enhances rAAV gene transfer efficiency in a murine model of Duchenne muscular dystrophy. [Board No.0734]   #genethon #congress #ESGCT2024 #Board European Society of Gene and Cell Therapy Isabelle Richard Maxime Ferrand Sonia Albini, PhD

📢 [ESGCT congress] 🫵SAVE THE DATE: Today from 06:00 pm - 07:30 pm CEST time- Don’t miss Martina Marinello from Genethon. She will present her poster: Dose-finding and toxicological studies of intravenous administration of an AAV9-ASAH1 vector for Farber disease and spinal muscular atrophy with progressive epilepsy.[Board No.0890] #genethon #congress #ESGCT2024 #Board European Society of Gene and Cell Therapy Ana Buj Bello

📢 [ESGCT congress] 🫵SAVE THE DATE: Today from 06:00 pm - 07:30 pm CEST time- Come and meet ALEXANDRA TACHTSIDI from Genethon with her poster: CRISPR/Cas9-induced genomic alterations and AAV targeted integration in HSPC through the lens of Long Read-sequencing, [Board No.0602] #genethon #congress #ESGCT2024 #Board European Society of Gene and Cell Therapy Mario Amendola Isabelle Richard

📢 [ESGCT congress] 🫵SAVE THE DATE: Today from 06:00 pm - 07:30 pm CEST time- Come and meet Amandine FRANÇOIS from Genethon, with her poster: A novel AAV gene therapy strategy targeting the liver to treat bone and dental defects in X-linked hypophosphatemia, [Board No.1010] #genethon #congress #ESGCT2024 #Board European Society of Gene and Cell Therapy Fanny Collaud Giuseppe Ronzitti

Well done and thank you to our Genethon “runners” for taking up the challenge of the first European Society of Gene and Cell Therapy FUN RUN at 7am despite the rain! Anne GOBERT Coralie MAIZERAY Séverine Pouillot LAETITIA PRZYBYLSKI-NICAISE Fanny Collaud Céline Labetoule

📢 [ESGCT congress] 🫵SAVE THE DATE - Today from 03:30 pm - 05:30 pm CEST time. Don’t miss the oral presentation about in vivo Gene Editing in Preclinical Models: “CRISPR-Cas9 mediated endogenous utrophin upregulation improves Duchenne Muscular Dystrophy “ by Maëlle RALU, PhD Student Genethon. #genethon #congress #ESGCT2024 #presentation #gene editing #DMD Mario Amendola European Society of Gene and Cell Therapy 

📢 [ESGCT congress] 🫵SAVE THE DATE: Today from 02:00 pm - 03:30 pm CEST time - Don’t miss Jérôme DENARD from Genethon with his poster: Long-term correction of acid ceramidase deficiency by intravenous AAV-mediated gene therapy in presymptomatic P361R-Farber mice, [Board No.0893] #genethon #congress #ESGCT2024 #Board European Society of Gene and Cell Therapy Mario Amendola Ana Buj Bello  

👏 🙌 Congratulations to Edith Renaud-Gabardos and Maëlle RALU for obtaining one of the highest-rated #abstracts and receiving the Travel Grant ! Ana Buj Bello Mario Amendola