Today, I’m thrilled have our lab’s work featured on the cover of
@Nature
In this article, we have developed a therapeutic approach for a severe condition called Timothy syndrome. This has been a long journey. It started with making hiPS cell-derived neurons in 2D cultures 15 years ago when I was a postdoc and continued with later creating in the lab
#organoid and then
#assembloid models of this disease caused by a mutation in a calcium channel.
Over time, these human cellular models and the biological insights we gained enable us to design a therapeutic strategy, which we validated in vivo following circuit integration of patient-derived organoids into the rat brain.
Grateful for the incredible work led by
@XiaoyuChenxy and
@fikribirey in the lab! And the many close collaborators who participated to this line of research over the years.