Therapeutic reversal of Huntington’s disease by in vivo self-assembled siRNAs
Open accessAbstract
Huntington’s disease is an autosomal-dominant neurodegenerative disease caused by CAG expansion in exon 1 of the huntingtin (HTT) gene. Since mutant huntingtin (mHTT) protein is the root cause of Huntington’s disease, oligonucleotide-based therapeutic approaches using small interfering RNAs (siRNAs) and antisense oligonucleotides designed to specifically silence mHTT may be novel therapeutic strategies for Huntington’s disease. Unfortunately, the lack of an effective in vivo delivery system remains a major obstacle to realizing the full potential of oligonucleotide therapeutics, especially regarding the delivery of oligonucleotides to the cortex and striatum, the most severely affected brain regions in Huntington’s disease. In this study, we present a synthetic biology strategy that integrates the naturally existing exosome-circulating system with artificial genetic circuits for self-assembly and delivery of mHTT-silencing siRNA to the cortex and striatum. We designed a cytomegalovirus promoter-directed genetic circuit encoding both a neuron-targeting rabies virus glycoprotein tag and an mHTT siRNA. After being taken up by mouse livers after intravenous injection, this circuit was able to reprogramme hepatocytes to transcribe and self-assemble mHTT siRNA into rabies virus glycoprotein-tagged exosomes. The mHTT siRNA was further delivered through the exosome-circulating system and guided by a rabies virus glycoprotein tag to the cortex and striatum. Consequently, in three mouse models of Huntington’s disease treated with this circuit, the levels of mHTT protein and toxic aggregates were successfully reduced in the cortex and striatum, therefore ameliorating behavioural deficits and striatal and cortical neuropathologies. Overall, our findings establish a convenient, effective and safe strategy for self-assembly of siRNAs in vivo that may provide a significant therapeutic benefit for Huntington’s disease.
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A genetic circuit encoding a neuron-targeting RVG tag and mHTT siRNA self-assembled into EVs in vivo, reducing mHTT levels in the cortex and striatum.
“a cytomegalovirus promoter-directed genetic circuit encoding a neuron-targeting RVG tag and a mHTT siRNA was injected into mice, uptaken by liver cells, and self-assembled into EVs. The endogenous EV-circulating system delivered the engineered EVs to the brain, reducing the levels of mHTT in the cortex and striatum.”
From Extracellular vesicles as nanotheranostic platforms for targeted neurological disorder interventions (2024) · cites this paper as [281]
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Self-citation
- Authors
- Zhang, L., Wu, T., Shan, Y., Li, G., Ni, X., Chen, X., Hu, X., Lin, L., Li, Y., Guan, Y., Gao, J., Chen, D., Zhang, Y., Pei, Z., & Chen, X.
- Affiliations
- Chinese Academy of Medical Sciences & Peking Union Medical College (CN)
- Sun Yat-sen University (CN)
- Guangdong Laboratory Animals Monitoring Institute (CN)
- Guangdong Pharmaceutical University (CN)
- Corresponding author
- Pei, Zhong
- Chen, Xi
- ORCID
- Venue
- Brain
- Publisher
- Oxford University Press (OUP)
- Published
- 2021-11-01
- Volume
- 144
- Issue
- 11
- Pages
- 3421-3435
- ISSN
- 0006-8950 (ISSN-L)
- 1460-2156
- PMC
- PMC8677541
- OpenAlex
- W4200306678
- Cited by
- 97
- References
- 65
- Citations by year
- 2026: 17 · 2025: 25 · 2024: 25 · 2023: 21 · 2022: 8 · 2021: 1
- FWCI
- 4.32
- Type
- journal article
- Language
- en
- Open access
- hybrid
- doi.org
- Licence
- Locations
- Brain (publishedVersion, cc-by-nc)
- PubMed Central (publishedVersion, cc-by-nc)
- PubMed (publishedVersion)
- Funders
- National Natural Science Foundation of China (32022015, 31871295)
- National Key Research and Development Program of China, Stem Cell and Translational Research (2017YFA0105104)
- Natural Science Foundation of Jiangsu Province (BE2016737)
- Guangdong Provincial Science and Technology Plan Project (2016B030230002)
- Guangdong Provincial Key Laboratory for Diagnosis and Treatment of Major Neurological Diseases (2017B030314103)
- Fundamental Research Funds for the Central Universities (020814380162)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Life Sciences › Neuroscience › Cellular and Molecular Neuroscience › Genetic Neurodegenerative Diseases
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › Extracellular vesicles in disease
- Keywords
- Huntington's disease, mutant huntingtin, exosomes, rabies virus glycoprotein, hepatocyte reprogramming, in vivo gene delivery, CAG repeat expansion, oligonucleotide therapeutics, striatum, cortex, neurodegeneration
- MeSH terms
- Huntingtin Protein, Animals, Genetic Engineering, Huntington Disease, Liver, Transfection, Genetic Therapy, RNA, Small Interfering, Mice, Exosomes
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