In studying genetic diseases, we learn that we should first find the gene, then find the associated protein function, and finally work on curing the disease. But science is not so linear. In the case of Batten disease, a group of rare childhood lysosomal storage brain diseases, the race to a “cure” has outpaced the understanding of the underlying cause of the disease.
In this talk, I will describe our contributions to gene therapy for CLN5 and CLN6 Batten disease, and the development of new iPSC-derived neuronal models to understand the biology of the associated proteins.
|Date||Tuesday, 4 May 2021|
|Time||12:00pm - 1:00pm|
|Event Category||Health Sciences|
|Location||Biochemistry Seminar Room BIG13, University of Otago, Dunedin|
|Contact Name||Department of Biochemistry|