期刊
ALZHEIMERS RESEARCH & THERAPY
卷 9, 期 -, 页码 -出版社
BMC
DOI: 10.1186/s13195-017-0307-1
关键词
Gene suppression; RNA interference; Anti-sense oligonucleotides; Zinc-finger proteins; CRISPR/Cas9; Therapeutics; Huntington's disease; Dementia
资金
- Medical Research Council UK Clinical Research Fellowship
- EU
- Medical Research Council UK
- CHDI Foundation
- Huntington Disease Association of the UK
- European Huntington's Disease Network
- Wellcome Trust
- Rosetrees Trust
- Takeda Pharmaceuticals
- MRC [MR/K023268/1] Funding Source: UKRI
- Medical Research Council [MR/K023268/1, MR/P007015/1] Funding Source: researchfish
- Rosetrees Trust [M574] Funding Source: researchfish
- Wellcome Trust [200181/Z/15/Z] Funding Source: researchfish
Gene suppression approaches have emerged over the last 20 years as a novel therapeutic approach for the treatment of neurodegenerative diseases. These include RNA interference and anti-sense oligonucleotides, both of which act at the post-transcriptional level, and genome-editing techniques, which aim to repair the responsible mutant gene. All serve to inhibit the expression of disease-causing proteins, leading to the potential prevention or even reversal of the disease phenotype. In this review we summarise the main developments in gene suppression strategies, using examples from Huntington's disease and other inherited causes of neurodegeneration, and explore how these might illuminate a path to tackle other proteinopathy-associated dementias in the future.
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