Decoding and Repairing Motor Neuron Disease with CRISPR and iPS cells

Bruce Conklin, Ph.D.

Seminar Information

Seminar Date
October 15, 2021 - 2:00 PM

The FUNG Auditorium

Bruce Conklin

Speaker Bio

Bruce R Conklin MD is a Senior Investigator at the Gladstone Institutes and a Professor at UCSF.
Conklin is also the Deputy Director of the Innovative Genomics Institute founded by Jennifer Doudna, Ph.D. The Conklin Lab focuses on biomedical applications of CRISPR technology with an emphasis on motor neuron diseases such as ALS. Decoding human genetic disease allows Dr. Conklin to develop models of the pathology that can be directly tested with gene correction or targeted drug therapy. Dominant negative mutations are particularly promising therapeutic targets since they are resistant to traditional therapies, yet precise excision of disease-causing alleles could provide a cure.
Dr. Conklin is using patient-derived induced pluripotent stem cells (iPSCs) to model cells such as motor neurons. Developing CRISPR genome surgery in human neurons provides a path to devise improved cellular models as well as human therapies.