Embracing Heterogeneity in ALS to Develop Diverse In Vitro Models that can Support Development of Targeted Therapeutics
Time: 11:10 am
day: Conference Day One Track A
Details:
• ALS patients present with significant heterogeneity in disease features, that may share related, common mechanisms
• Most ALS disease models focus on rare forms of the disease and are not genetically representative of >80% of patients with sporadic ALS
• Developing and selecting drug candidates in diverse patient cell lines is standard practice in oncology therapeutic discovery
• Development of a large panel of patient models and iPSC lines has facilitated the rapid discovery and development of UNC13A targeting antisense oligonucleotide (ASO) therapeutics
