“Engineered zinc finger protein transcription factors potently reduce brain PrP expression and extend survival in prion-infected mice”
Bryan Zeitler et al.
This is a post-conference recording of a presentation given at the Prion 2022 conference on September 16, 2022 by Bryan Zeitler, Senior Director, Gene Regulation at Sangamo Therapeutics.
Nina Oberbeck from Gate Bioscience at the 2023 CJD Conference, Melbourne Australia.
Post by Eric Minikel from Prion Alliance
Link – First details of Ionis trial disclosed
Spanish version link- Primeros detalles revelados sobre el ensayo de Ionis
Please find below links to some general information on clinical trials which may be helpful in the lead-up to the commencement of clinical trials for prion disease:
U.S. Department of Health & Human Services – Participating in Human Research
You may find the videos to be very approachable, and they have a very handy PDF with questions that people should consider asking a study investigator. Many of their resources are available in English and Spanish, which is a nice bonus!
U.S. Food & Drug Administration – Clinical Trials: What Patients Need to Know
Explanations are very basic, but include special pages on Informed Consent and explain the difference between a trial and medical treatment which is very helpful, especially for communities like ours that are relatively new to trials.
U.S. National Institute of Health – Clinical Research Trials and You
This site has some great tools as well, although they may be a little bit more technical that the Health & Human Services ones.
John Collinge (MRC Prion Unit, University College London, London, UK) discusses a first-in-human treatment programme to give PRN100, an anti-prion-protein monoclonal antibody, to patients with Creutzfeldt–Jakob disease; the report is published in the April issue of The Lancet Neurology.
In Conversation with… John Collinge (link to podcast with listing options)