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Recent Advances in Gene Therapy for Familial Hypercholesterolemia: An Update Review.
| Content Provider | Europe PMC |
|---|---|
| Author | Fu, Qingan Hu, Lijuan Shen, Tianzhou Yang, Renqiang Jiang, Long |
| Editor | Kaminski, Karol |
| Copyright Year | 2022 |
| Abstract | (1) Background: Existing lipid-lowering therapies have difficulty in achieving lipid target levels in patients with familial hypercholesterolemia (FH), especially in the treatment of patients with homozygous familial hypercholesterolemia. (2) Method: All of the literature data containing "Familial hypercholesterolemia" and "Gene Therapy" in PubMed and Clinical Trials from 2018 to 2022 were selected. (3) Results: The rapid development of gene therapy technology in recent years is expected to change the treatment status of FH patients. As emerging gene therapy vectors, the optimized adeno-associated viruses, exosomes, and lipid nanoparticles have demonstrated an improved safety and higher transfection efficiency. Various RNA-targeted therapies are in phase 1-3 clinical trials, such as small interfering RNA-based drugs inclisiran, ARO-ANG3, ARO-APOC3, olpasiran, SLN360, and antisense oligonucleotide-based drugs AZD8233, vupanorsen, volanesorsen, IONIS-APO(a)Rx, etc., all of which have demonstrated excellent lipid-lowering effects. With gene editing technologies, such as CRISPR-Cas 9 and meganuclease, completing animal experiments in mice or cynomolgus monkeys and demonstrating lasting lipid-lowering effects, patients with FH are expected to reach a permanent cure in the future. (4) Conclusion: Gene therapy is being widely used for the lipid-lowering treatment of FH patients and has shown excellent therapeutic promise, but the current delivery efficiency, economic burden, immunogenicity and the precision of gene therapy can be further optimized. |
| Journal | Journal of Clinical Medicine [J Clin Med] |
| Volume Number | 11 |
| DOI | 10.3390/jcm11226773 |
| PubMed Central reference number | PMC9699383 |
| Issue Number | 22 |
| PubMed reference number | 36431249 |
| e-ISSN | 20770383 |
| Language | English |
| Publisher | Molecular Diversity Preservation International (MDPI) |
| Publisher Date | 2022-11-16 |
| Access Restriction | Open |
| Subject Keyword | familial hypercholesterolemia antisense oligonucleotide small interfering RNA CRISPR/Cas9 gene therapy |
| Content Type | Text |
| Resource Type | Article |
| Subject | Medicine |
