In this 2021 course, Dr. Ignacio Munoz-Sanjuan, VP of translational biology at CHDI Foundation, speaks to the clinicians and movement disorder residents as part of a course about Huntington’s disease organized by EHDN and MDS.
The seminar focuses on our current understanding of HTT biological aspects relevant to the development of therapeutics targeting the cause of the disease, the mutant Huntingtin (HTT) gene, via various approaches, including small molecules, antisense oligonucleotides (ASOs) and mRNA-targeting gene therapy agents delivered via adeno-associated viruses (AAVs).
Ignacio describes current biological, technological and medical challenges in conducting mHTT lowering therapeutic studies, and focuses on the present and future of this most essential research area for developing effective therapies for HD. It is a course full of important information about ongoing trials, and work to demonstrate that this approach is likely to be efficacious in slowing disease progression,
The talk has been approved for public disclosure by MDS and EHDN via the Factor-H website, and for this we remain grateful.
Sharing information is critical to maintain the HD community informed and active.