Spotlight on Gene Therapy: finding a Cure

Our lab attended the annual meeting of the HPS Network.

HPS stands for Hermansky-Pudlak syndrome. It is a rare genetic disorder that causes albinism, visual impairment, and platelet dysfunction that causes bleeding issues. It has been identified in individuals worlwide, but has a very high prevalence in Puerto Rico due to a founder effect.

The attendees of the "Meeting of the Minds" at the 2017 HPS Network Meeting.

Dr Mueller joined the HPS Network in 2016, in a group efforts of all scientists involved in HPS research to develop therapies for HPS patients, and in particular gene therapy. Saturday was a day-long scientific meeting, where all attendees presented their latest, unpublished research. On Sunday, scientists gave presentations to the HPS community. This year again, Dr Mueller presented on gene therapy, what it is, how it works, and what it may have to offer for HPS patients. He also answered questions from the patients, who this year were curious to learn more about CRISPR and genome editing.

The lab is looking forward to doing some more collaborative work with other HPS researchers!