A non-viral CRISPR/Cas9 delivery system for therapeutically targeting HBV DNA and pcsk9 in vivo
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Lipid nanoparticle-mRNA formulations have been applied to produce therapeutic proteins or antibodies, to edit virus genomes and to engineer immune cells.
“In addition, lipid nanoparticle–mRNA formulations have been applied to produce therapeutic proteins or antibodies39,75,145,146, to edit virus genomes105 and to engineer immune cells136.”
From Lipid nanoparticles for mRNA delivery (2021) · cites this paper as [105]
Gene-editing components can be delivered by lipid nanoparticle-mRNA formulations to treat genetic diseases, including hereditary tyrosinaemia type I, hypercholesterolaemia, lipoprotein metabolism disorders and transthyretin amyloidosis, as demonstrated in preclinical studies.
“Gene-editing components can be delivered by lipid nanoparticle–mRNA formulations to treat genetic diseases, including HTI92,238, hypercholesterolaemia81,96,105,239, lipoprotein metabolism disorders85 and transthyretin amyloidosis239,240, which has been demonstrated in preclinical studies.”
From Lipid nanoparticles for mRNA delivery (2021) · cites this paper as [105]
After i.v. injection of Cas9 mRNA TT3 LNPs following sgRNA TT3 LNPs, the PCSK9 protein level was remarkably decreased in C57BL/6 mice.
“After i.v. injection of Cas9 mRNA TT3 LNPs following the injection of sgRNA TT3 LNPs, the PCSK9 protein level was remarkably decreased in C57BL/6 mice.[139]”
From RNA delivery biomaterials for the treatment of genetic and rare diseases (2019) · cites this paper as [139]
Jiang et al.'s lipid-like nanoparticles achieved disruption of HBV DNA and pcsk9 gene in vivo.
“Jiang et al. developed lipid-like nanoparticles for targeted delivery of Cas9 mRNA and sgRNA to the liver. This system achieved disruption of HBV DNA and the proprotein convertase subtilisin/kexin type 9 (pcsk9) gene in vivo [104].”
From Non-viral delivery systems for CRISPR/Cas9-based genome editing: challenges and opportunities (2018) · cites this paper as [104]
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- Authors
- Jiang, C., Mei, M., Li, B., Zhu, X., Zu, W., Tian, Y., Wang, Q., Guo, Y., Dong, Y., & Tan, X.
- Affiliations
- Center for Life Sciences (CN)
- The Ohio State University (US)
- Tsinghua University (CN)
- Corresponding author
- Dong, Yizhou
- Tan, Xu
- ORCID
- Venue
- Cell Research
- Published
- 2017-01-24
- Volume
- 27
- Issue
- 3
- Pages
- 440-443
- ISSN
- 1001-0602 (ISSN-L)
- 1748-7838
- PMC
- PMC5339835
- OpenAlex
- W2582215039
- Cited by
- 295
- References
- 15
- Citations by year
- 2026: 8 · 2025: 15 · 2024: 24 · 2023: 34 · 2022: 49 · 2021: 35 · 2020: 37 · 2019: 36 · 2018: 40 · 2017: 17
- FWCI
- 14.53
- Type
- journal article
- Language
- en
- Open access
- bronze
- doi.org
- Licence
- www.springer.com/tdm (tdm)
- Locations
- Cell Research (publishedVersion)
- Europe PMC (PubMed Central) (publishedVersion)
- PubMed Central (publishedVersion)
- PubMed (publishedVersion)
- Funders
- Bill and Melinda Gates Foundation
- Ohio State University
- National PKU Alliance
- National Institute of General Medical Sciences (R35GM119679)
- Topics
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › RNA Interference and Gene Delivery
- Life Sciences › Biochemistry, Genetics and Molecular Biology › Molecular Biology › CRISPR and Genetic Engineering
- Physical Sciences › Materials Science › Biomaterials › Nanoparticle-Based Drug Delivery
- Keywords
- CRISPR/Cas9, non-viral gene delivery, PCSK9, hypercholesterolemia, liver-targeted drug delivery, single guide RNA, in vivo gene editing
- MeSH terms
- Proprotein Convertase 9, CRISPR-Associated Protein 9, Animals, Base Sequence, DNA, Viral, Hepatitis B virus, Humans, Lipids, Liver, Mice, Inbred C57BL, Gene Targeting, NIH 3T3 Cells, Mice, Nanoparticles, HEK293 Cells, CRISPR-Cas Systems
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