Recent Advances in Gene Therapy for Familial Hypercholesterolemia: An Update Review
(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 h...
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| Format: | Article |
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MDPI AG
2022-11-01
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| Series: | Journal of Clinical Medicine |
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| Online Access: | https://www.mdpi.com/2077-0383/11/22/6773 |
| _version_ | 1797464916543668224 |
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| author | Qingan Fu Lijuan Hu Tianzhou Shen Renqiang Yang Long Jiang |
| author_facet | Qingan Fu Lijuan Hu Tianzhou Shen Renqiang Yang Long Jiang |
| author_sort | Qingan Fu |
| collection | DOAJ |
| description | (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. |
| first_indexed | 2024-03-09T18:14:56Z |
| format | Article |
| id | doaj.art-a13b8fc6a02c4ed394575c973473081f |
| institution | Directory Open Access Journal |
| issn | 2077-0383 |
| language | English |
| last_indexed | 2024-03-09T18:14:56Z |
| publishDate | 2022-11-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | Journal of Clinical Medicine |
| spelling | doaj.art-a13b8fc6a02c4ed394575c973473081f2023-11-24T08:49:52ZengMDPI AGJournal of Clinical Medicine2077-03832022-11-011122677310.3390/jcm11226773Recent Advances in Gene Therapy for Familial Hypercholesterolemia: An Update ReviewQingan Fu0Lijuan Hu1Tianzhou Shen2Renqiang Yang3Long Jiang4. Department of Cardiovascular Medicine, the Second Affiliated Hospital of Nanchang University, Nanchang 330006, China. Department of Nursing, Nanchang Medical College, Nanchang 330052, China. Department of Cardiovascular Medicine, the Second Affiliated Hospital of Nanchang University, Nanchang 330006, China. Department of Cardiovascular Medicine, the Second Affiliated Hospital of Nanchang University, Nanchang 330006, China. Department of Cardiovascular Medicine, the Second Affiliated Hospital of Nanchang University, Nanchang 330006, China(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.https://www.mdpi.com/2077-0383/11/22/6773familial hypercholesterolemiaantisense oligonucleotidesmall interfering RNACRISPR/Cas9gene therapy |
| spellingShingle | Qingan Fu Lijuan Hu Tianzhou Shen Renqiang Yang Long Jiang Recent Advances in Gene Therapy for Familial Hypercholesterolemia: An Update Review Journal of Clinical Medicine familial hypercholesterolemia antisense oligonucleotide small interfering RNA CRISPR/Cas9 gene therapy |
| title | Recent Advances in Gene Therapy for Familial Hypercholesterolemia: An Update Review |
| title_full | Recent Advances in Gene Therapy for Familial Hypercholesterolemia: An Update Review |
| title_fullStr | Recent Advances in Gene Therapy for Familial Hypercholesterolemia: An Update Review |
| title_full_unstemmed | Recent Advances in Gene Therapy for Familial Hypercholesterolemia: An Update Review |
| title_short | Recent Advances in Gene Therapy for Familial Hypercholesterolemia: An Update Review |
| title_sort | recent advances in gene therapy for familial hypercholesterolemia an update review |
| topic | familial hypercholesterolemia antisense oligonucleotide small interfering RNA CRISPR/Cas9 gene therapy |
| url | https://www.mdpi.com/2077-0383/11/22/6773 |
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