Gene Therapy Strategies For Myotonic Dystrophy Type 1

Autor: Beatrice Cardinali 1, Claudia Provenzano 1, Mariapaola Izzo 1, Jonathan Battistini 1, Georgios Strimpakos 1, Elisabetta Golini 1, Silvia Mandillo*1, Ferdinando Scavizzi 1, Marcello Raspa 1, Christine Voellenkle 2, Alessandra Perfetti 2, Denisa Baci 2, Fabio Martelli 2, Genevieve Gourdon 3, Germana Falcone 1

Publikováno v: "Mechanistic Insights into Neurological Disorders and New Therapeutic Strategies", DSB Conference, CNR Rome, Italy, 07-08/07/2021
info:cnr-pdr/source/autori:Beatrice Cardinali 1, Claudia Provenzano 1, Mariapaola Izzo 1, Jonathan Battistini 1, Georgios Strimpakos 1, Elisabetta Golini 1, Silvia Mandillo*1, Ferdinando Scavizzi 1, Marcello Raspa 1, Christine Voellenkle 2, Alessandra Perfetti 2, Denisa Baci 2, Fabio Martelli 2, Genevieve Gourdon 3, Germana Falcone 1/congresso_nome:"Mechanistic Insights into Neurological Disorders and New Therapeutic Strategies"/congresso_luogo:DSB Conference, CNR Rome, Italy/congresso_data:07-08%2F07%2F2021/anno:2021/pagina_da:/pagina_a:/intervallo_pagine

Background: Myotonic dystrophy type 1 (DM1) is a dominantly inherited neuromuscular disease caused by the abnormal expansion of CTG-triplets in the 3' untranslated region of the DMPK gene. While therapeutic approaches that neutralize the toxic DMPK t

Externí odkaz: https://explore.openaire.eu/search/publication?articleId=cnr_________::01b75d694032588d55600578e19c1ada
https://publications.cnr.it/doc/458133