The journey from discovering and developing effective, precise medications to using them correctly and safely in patients is hardly fast and easy. Nor is it a straight shot. Scientists in the UCSF School of Pharmacy are challenging the status quo every step of the way.
Researchers have demonstrated the ability to program groups of individual cells to self-organize into multi-layered structures reminiscent of simple organisms or the first stages of embryonic development.
Organoids serve as dioramas of disease, allowing UCSF scientists to understand how and why problems occur during tissue development. It's also a small step toward the creation of full-sized organs we could use for transplant.
A nationwide project that includes two UCSF researchers will use the latest technology, including gene editing, to gain insights into human biology that could one day lead to treatments for complex genetic diseases.