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Learn about the process of clinical research and drug development and the role rare disease patient communities can play in the process from this recording of the 2022 Virtual Conference Session presented by Laura Iliescu, MSc, Director, Patient Advocacy Strategy, Center for Rare Diseases.
Learn about antisense oligonucleotide targeting of PRNP mRNA as a potential treatment for prion disease from this recording of the 2020 Virtual Conference Session presented by Anne Smith, PhD, Executive Director of Clinical Development at Ionis Pharmaceuticals, a California-based biotechnology company focused on RNA-targeting therapeutics.
Human prion diseases, including Creutzfeldt–Jakob disease (CJD), are rapidly progressive, invariably fatal neurodegenerative conditions with no effective therapies. Their pathogenesis involves the obligate recruitment of cellular prion protein (PrPC) into self-propagating multimeric assemblies or prions. Preclinical studies have firmly validated the targeting of PrPC as a therapeutic strategy. Read a study in The Lancet Neurology that evaluates a first-in-human treatment programme using an anti-PrPC monoclonal antibody under a Specials Licence.
Learn about Prion protein monoclonal antibody (PRN100) therapy for CJD from this recording of the 2022 Virtual Conference Session presented by Prof John Collinge, Professor of Neurology, MRC Prion Unit at UCL, UCL Institute of Prion Diseases.
Learn about a potential future treatment for prion disease from this recording of the 2022 presentation by Brian Zeitler, Senior Director of Gene Regulation, Sangamo Therapeutics: “Engineered zinc finger protein transcription factors potently reduce brain PrP expression and extend survival in prion-infected mice”
Researchers evaluated a novel epigenetic regulation approach using Zinc Finger Repressors (ZFRs) to ablate PrP expression at the transcriptional level. When delivered using adeno-associated virus (AAV), ZFRs potently and specifically reduced prion mRNA expression by >95% in vitro and to near undetectable levels within single neurons in vivo. In wildtype mice, ZFRs stably lowered neuronal PrP expression throughout the central nervous system for at least 17 months. In mice inoculated with misfolded PrP, AAV-ZFRs given at either early or late disease stages profoundly extended lifespan, significantly reduced PrP in the brain, and improved an array of molecular, histological, biomarker, and behavioral readouts.
This website was made possible by a generous donation from Cookie Stivison, in memory of her husband Tom Stivison, and a grant from the Centers for Disease Control and Prevention.
