Gene transfer and genome editing for familial hypercholesterolemia
Familial hypercholesterolemia (FH) is an autosomal dominant inherited disease characterized by high circulating low-density lipoprotein (LDL) cholesterol. High circulating LDL cholesterol in FH is due to dysfunctional LDL receptors, and is mainly expressed by hepatocytes. Affected patients rapidly d...
| Main Authors: | , |
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| Format: | Article |
| Language: | English |
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Frontiers Media S.A.
2023-04-01
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| Series: | Frontiers in Molecular Medicine |
| Subjects: | |
| Online Access: | https://www.frontiersin.org/articles/10.3389/fmmed.2023.1140997/full |
| _version_ | 1797853694175215616 |
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| author | Cesare Canepari Cesare Canepari Alessio Cantore Alessio Cantore |
| author_facet | Cesare Canepari Cesare Canepari Alessio Cantore Alessio Cantore |
| author_sort | Cesare Canepari |
| collection | DOAJ |
| description | Familial hypercholesterolemia (FH) is an autosomal dominant inherited disease characterized by high circulating low-density lipoprotein (LDL) cholesterol. High circulating LDL cholesterol in FH is due to dysfunctional LDL receptors, and is mainly expressed by hepatocytes. Affected patients rapidly develop atherosclerosis, potentially leading to myocardial infarction and death within the third decade of life if left untreated. Here, we introduce the disease pathogenesis and available treatment options. We highlight different possible targets of therapeutic intervention. We then review different gene therapy strategies currently under development, which may become novel therapeutic options in the future, and discuss their advantages and disadvantages. Finally, we briefly outline the potential applications of some of these strategies for the more common acquired hypercholesterolemia disease. |
| first_indexed | 2024-04-09T19:53:47Z |
| format | Article |
| id | doaj.art-03fdeca755b249ddbf0b14a4f39c2a3b |
| institution | Directory Open Access Journal |
| issn | 2674-0095 |
| language | English |
| last_indexed | 2024-04-09T19:53:47Z |
| publishDate | 2023-04-01 |
| publisher | Frontiers Media S.A. |
| record_format | Article |
| series | Frontiers in Molecular Medicine |
| spelling | doaj.art-03fdeca755b249ddbf0b14a4f39c2a3b2023-04-03T05:32:34ZengFrontiers Media S.A.Frontiers in Molecular Medicine2674-00952023-04-01310.3389/fmmed.2023.11409971140997Gene transfer and genome editing for familial hypercholesterolemiaCesare Canepari0Cesare Canepari1Alessio Cantore2Alessio Cantore3San Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, ItalyVita-Salute San Raffaele University, Milan, ItalySan Raffaele Telethon Institute for Gene Therapy (SR-Tiget), IRCCS San Raffaele Scientific Institute, Milan, ItalyVita-Salute San Raffaele University, Milan, ItalyFamilial hypercholesterolemia (FH) is an autosomal dominant inherited disease characterized by high circulating low-density lipoprotein (LDL) cholesterol. High circulating LDL cholesterol in FH is due to dysfunctional LDL receptors, and is mainly expressed by hepatocytes. Affected patients rapidly develop atherosclerosis, potentially leading to myocardial infarction and death within the third decade of life if left untreated. Here, we introduce the disease pathogenesis and available treatment options. We highlight different possible targets of therapeutic intervention. We then review different gene therapy strategies currently under development, which may become novel therapeutic options in the future, and discuss their advantages and disadvantages. Finally, we briefly outline the potential applications of some of these strategies for the more common acquired hypercholesterolemia disease.https://www.frontiersin.org/articles/10.3389/fmmed.2023.1140997/fullgene therapyfamilial hypercholesterolemiagene editinglipoproteinslipid-lowering medications |
| spellingShingle | Cesare Canepari Cesare Canepari Alessio Cantore Alessio Cantore Gene transfer and genome editing for familial hypercholesterolemia Frontiers in Molecular Medicine gene therapy familial hypercholesterolemia gene editing lipoproteins lipid-lowering medications |
| title | Gene transfer and genome editing for familial hypercholesterolemia |
| title_full | Gene transfer and genome editing for familial hypercholesterolemia |
| title_fullStr | Gene transfer and genome editing for familial hypercholesterolemia |
| title_full_unstemmed | Gene transfer and genome editing for familial hypercholesterolemia |
| title_short | Gene transfer and genome editing for familial hypercholesterolemia |
| title_sort | gene transfer and genome editing for familial hypercholesterolemia |
| topic | gene therapy familial hypercholesterolemia gene editing lipoproteins lipid-lowering medications |
| url | https://www.frontiersin.org/articles/10.3389/fmmed.2023.1140997/full |
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