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Gene Therapy Strategies for Prophylactic and Therapeutic Treatments of Human Prion Diseases

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Prions and Diseases

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Abstract

Prion disease is a diverse family of fatal and usually transmissible and progressive neurodegenerative diseases that strike humans and many other mammal species, such as cattle, sheep, and cervids. The cellular PrP (PrPC) is the substrate for the replication of misfolded prion protein aggregates (PrPSc) that serve as the transmissible prion agents. PrPC is also essential for prion pathogenesis. No treatments are available for prion diseases. Numerous efforts with various anti-prion compounds or antibodies have not produced meaningful benefits for prion patients in clinical trials so far. The gene therapy technology has matured in the last several years and offers great hopes for effective treatment and prevention of prion diseases. Here, we review the current literature on prion gene therapy development and propose a few promising gene therapy strategies targeting various aspects of prion replication and pathogenesis.

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Authors and Affiliations

  1. Department of Pathology, Case Western Reserve University, Cleveland, OH, USA

    Manuel Camacho & Qingzhong Kong

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  1. Manuel Camacho
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  2. Qingzhong Kong
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Correspondence to Qingzhong Kong .

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Editors and Affiliations

  1. Departments of Pathology and Neurology, National Prion Disease Pathology Surveillance Center, National Center for Regenerative Medicine Case Western Reserve University, School of Medicine, Cleveland, OH, USA

    Wen-Quan Zou

  2. Department of Pathology, Case Western Reserve University School of Medicine, Cleveland, OH, USA

    Pierluigi Gambetti

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Camacho, M., Kong, Q. (2023). Gene Therapy Strategies for Prophylactic and Therapeutic Treatments of Human Prion Diseases. In: Zou, WQ., Gambetti, P. (eds) Prions and Diseases. Springer, Cham. https://doi.org/10.1007/978-3-031-20565-1_36

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