Work

Progress and Challenges in RNA Interference Therapy for Huntington Disease

Year: 2009

Type: article

Abstract: Huntington disease is an incurable, dominant neurodegenerative disorder caused by polyglutamine repeat expansion in the huntingtin protein. Reducing mutant huntingtin expression may offer a treatment for Huntington disease. RNA interference has emerged as a powerful method to silence dominant disease genes. As such, it is being developed as a prospective Huntington disease therapy. Here I discuss the current progress and important remaining challenges of RNA interference therapy for Huntington disease.

Source: Archives of Neurology

DOI: 10.1001/archneurol.2009.180

Author: Scott Q. Harper

Institutions: The Ohio State University Wexner Medical Center, Abigail Wexner Research Institute, The Ohio State University

Language: English

Topic: Genetic Neurodegenerative Diseases

Subfield: Cellular and Molecular Neuroscience

Field: Neuroscience

Domain: Life Sciences

SDG: Good health and well-being

Open Access status: bronze

OpenAlex ID: W2052423145