Title |
Delivering a disease-modifying treatment for Huntington's disease
|
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Published in |
Drug Discovery Today, January 2015
|
DOI | 10.1016/j.drudis.2014.09.011 |
Pubmed ID | |
Authors |
Bruno M.D.C. Godinho, Meenakshi Malhotra, Caitriona M. O’Driscoll, John F. Cryan |
Abstract |
Huntington's disease (HD) is an incurable genetic neurodegenerative disorder that leads to motor and cognitive decline. It is caused by an expanded polyglutamine tract within the Huntingtin (HTT) gene, which translates into a toxic mutant HTT protein. Although no cure has yet been discovered, novel therapeutic strategies, such as RNA interference (RNAi), antisense oligonucleotides (ASO), ribozymes, DNA enzymes, and genome-editing approaches, aimed at silencing or repairing the mutant HTT gene hold great promise. Indeed, several preclinical studies have demonstrated the utility of such strategies to improve HD neuropathology and symptoms. In this review, we critically summarise the main advances and limitations of each gene-silencing technology as an effective therapeutic tool for the treatment of HD. |
X Demographics
Geographical breakdown
Country | Count | As % |
---|---|---|
United States | 2 | 40% |
Ireland | 2 | 40% |
United Kingdom | 1 | 20% |
Demographic breakdown
Type | Count | As % |
---|---|---|
Members of the public | 3 | 60% |
Scientists | 1 | 20% |
Science communicators (journalists, bloggers, editors) | 1 | 20% |
Mendeley readers
Geographical breakdown
Country | Count | As % |
---|---|---|
United States | 4 | 3% |
Kazakhstan | 1 | <1% |
Unknown | 145 | 97% |
Demographic breakdown
Readers by professional status | Count | As % |
---|---|---|
Student > Bachelor | 31 | 21% |
Student > Master | 29 | 19% |
Researcher | 23 | 15% |
Student > Ph. D. Student | 15 | 10% |
Other | 7 | 5% |
Other | 18 | 12% |
Unknown | 27 | 18% |
Readers by discipline | Count | As % |
---|---|---|
Agricultural and Biological Sciences | 41 | 27% |
Biochemistry, Genetics and Molecular Biology | 30 | 20% |
Medicine and Dentistry | 16 | 11% |
Neuroscience | 15 | 10% |
Pharmacology, Toxicology and Pharmaceutical Science | 7 | 5% |
Other | 15 | 10% |
Unknown | 26 | 17% |