25 Sep SCN1A-UP! at the end – Gene therapy approaches
In this video, Dr Gaia Colasante summarizes the strategies and difficulties of developing gene therapy in Dravet Syndrome. She is project leader at IRCSS Ospedale San Raffaelle in Milan, Italy, and she manages a team committed to the development of gene therapy for the treatment of Dravet syndrome and other neurodevelopmental disorders.
She discussed her research results at the final meeting of the SCN1A-UP! project, a collaborative research involving six European research groups and five Patients Advocacy Organizations, which was funded by the EJPRD 2020..
In particular, the study, carried out on experimental models (cells and mice), focused on the identification of tools that could enable the healthy copy of the SCN1A gene to produce more of NAV1.1 protein (upregulation) to compensate for the deficiency caused by the malfunctioning of the gene. In addition, researchers paid special attention to the identification of viral vectors capable of spreading the gene therapy to the whole brain cells where the SCN1A gene is expressed.
Stay tuned for more information on the results of the study.
More information on the topic:
Know more about non-clinical development and experimental models
Project SCN1A-UP! – Therapeutic strategies for Dravet Syndrome