Accelerating Orphan Drug Discovery Using Zebrafish Models of Rare Diseases

سال انتشار: 1398
نوع سند: مقاله کنفرانسی
زبان: انگلیسی
مشاهده: 367

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شناسه ملی سند علمی:

RROYAN20_005

تاریخ نمایه سازی: 29 مهر 1398

چکیده مقاله:

There are approximately 7000 rare diseases, yet to date only a very limited number of orphan drugs are marketed. Develop-ing new drugs requires significant funds and time, and in the case of rare diseases, the commercial return is often insufficient for the pharmaceutical industry. For many rare diseases, drug repurposing would represent a faster, cheaper and less risky op-tion to identify drugs that would be of immediate therapeutic benefit to patients. A key obstacle currently in the way of a sys-tematic evaluation of approved drugs for their potential to treat a large number of different rare diseases is the availability of suitable screening models to perform drug repurposing screens.Because 80% of rare diseases are genetic in origin, it is possible to create animal models that have similar genetic defects and that mimic the human pathological conditions. Zebrafish, with their high genetic, physiological and pharmacological similar-ity with humans, offer the possibility of performing rapid drug repurposing screens using microscale, in vivo models of human diseases. To date, we have generated zebrafish models for a number of rare neurodevelopmental disorders, including Dravet syndrome, Lennox-Gasteaux syndrome, Otahara syndrome, Batten disease, and Zellweger syndrome. Drug repurposing and drug discovery screens using some of these zebrafish models has led to the identification of both approved drugs and new drug candidates, one of which will enter a Phase 2a clinical trial in 2020.

نویسندگان

ad Crawford

Norwegian University of Life Sciences, Faculty of Veterinary Medicine, Oslo, Norway . Tarbiat Modares University, Faculty of Agriculture, Tehran, and Faculty of Natural Resources & Marine Sciences, Noor, Iran . Institutes for Orphan Drug Discovery, Bremen