Blending In Vitro & In Vivo Models to Overcome Species Sequence Variation for Preclinical Testing of Human Genetic Targets

Time: 2:45 pm
day: Day One PM


• Differentiating motor neurons from human stem cells derived from ALS patients

• Creating a model system: implanting differentiated motor neurones into mouse brains

• Utilizing model system to investigate preclinical engagement of candidates against validated human genetic targets