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

Time: 2:40 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