CHDI Meeting in Palm Springs – Wednesday: Targeted Therapies, Clinical Trials and the Scientific Method

Wednesday kicked off with a session about huntingtin lowering therapies. The talks presented a series of different methods which aim to reduce the amount of deleterious polyQ expanded huntingtin in the cell including the hot CRISPR genome editing technology as well as exploiting protein degradation system in cells.

There was also an excellent poster session. 100+ posters, detailing work from a variety of scientific disciplines but all focussed on HD, were on display and their authors were on hand to discuss the data and ideas presented.

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A clinical trial “blitz” session ran a series of quickfire presentations from 4 different trials: SRX246, Rx CS1, PDE10A ICM and SIGNAL.

Marcus Munafo gave a lecture at the end of the day discussing the scientific method i.e. how we define a hypothesis, fairly test this and then interpret the data we get back from these tests. Marcus spoke of how scientists are inclined to bias and cherry picking data as they seek interesting outcomes in the pursuit of publications and grant funding, highlighting some extreme cases of scientific fraud. Marcus is also an advocate for open data, believing that by exposing raw data to the wider community, scientists are more likely to thoroughly check their data whilst not overstating or embellishing their conclusions.

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