Recent Advances in the Treatment of Huntington’s Disease: Targeting DNA and RNA
| Autor: | Kathleen M. Shannon |
|---|---|
| Rok vydání: | 2020 |
| Předmět: |
Genetic enhancement
Biology 03 medical and health sciences 0302 clinical medicine Genome editing Huntington's disease Mutant protein medicine Animals Humans Pharmacology (medical) RNA Small Interfering Gene Editing Oligonucleotide RNA DNA Genetic Therapy Oligonucleotides Antisense Polyglutamine tract medicine.disease 030227 psychiatry Psychiatry and Mental health Huntington Disease Neurology (clinical) Trinucleotide repeat expansion Neuroscience 030217 neurology & neurosurgery |
| Zdroj: | CNS Drugs. 34:219-228 |
| ISSN: | 1179-1934 1172-7047 |
| DOI: | 10.1007/s40263-019-00695-3 |
| Popis: | Huntington's disease is a dominantly inherited neurodegenerative disease caused by an unstable expanded trinucleotide repeat at the short end of the fourth chromosome. Central nervous system pathology begins in the striatum, eventually affecting the entire brain and occurs consequent to multiple intracellular derangements. The proximate cause is a mutant protein with an elongated polyglutamine tract. Pharmacological approaches targeting multiple domains of intracellular functions have universally been disappointing. However, recent developments in gene therapy, including antisense oligonucleotides, small interfering RNAs, and gene editing are bringing new hope to the Huntington's community. This review discusses the promises and challenges of these new potential treatments. |
| Databáze: | OpenAIRE |
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