Advances in Gene Therapy Techniques to Treat <i>LRRK2</i> Gene Mutation
Leucine-rich repeat kinase 2 (<i>LRRK2</i>) gene mutation is an autosomal dominant mutation associated with Parkinson’s disease (PD). Among <i>LRRK2</i> gene mutations, the <i>LRRK2</i> G2019S mutation is frequently involved in PD onset. Currently, diverse gene co...
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| Format: | Article |
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MDPI AG
2022-12-01
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| Series: | Biomolecules |
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| Online Access: | https://www.mdpi.com/2218-273X/12/12/1814 |
| _version_ | 1797461251150839808 |
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| author | Sun-Ku Chung Seo-Young Lee |
| author_facet | Sun-Ku Chung Seo-Young Lee |
| author_sort | Sun-Ku Chung |
| collection | DOAJ |
| description | Leucine-rich repeat kinase 2 (<i>LRRK2</i>) gene mutation is an autosomal dominant mutation associated with Parkinson’s disease (PD). Among <i>LRRK2</i> gene mutations, the <i>LRRK2</i> G2019S mutation is frequently involved in PD onset. Currently, diverse gene correction tools such as zinc finger nucleases (ZFNs), helper-dependent adenoviral vector (HDAdV), the bacterial artificial chromosome-based homologous recombination (BAC-based HR) system, and CRISPR/Cas9-homology-directed repair (HDR) or adenine base editor (ABE) are used in genome editing. Gene correction of the <i>LRRK2</i> G2019S mutation has been applied whenever new gene therapy tools emerge, being mainly applied to induced pluripotent stem cells (<i>LRRK2</i> G2019S-mutant iPSCs). Here, we comprehensively introduce the principles and methods of each programmable nuclease such as ZFN, CRISPR/Cas9-HDR or ABE applied to <i>LRRK2</i> G2019S, as well as those of HDAdV or BAC-based HR systems used as nonprogrammable nuclease systems. |
| first_indexed | 2024-03-09T17:17:44Z |
| format | Article |
| id | doaj.art-ed5ad9f1aa934f21a72496f28f5808a6 |
| institution | Directory Open Access Journal |
| issn | 2218-273X |
| language | English |
| last_indexed | 2024-03-09T17:17:44Z |
| publishDate | 2022-12-01 |
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| record_format | Article |
| series | Biomolecules |
| spelling | doaj.art-ed5ad9f1aa934f21a72496f28f5808a62023-11-24T13:33:48ZengMDPI AGBiomolecules2218-273X2022-12-011212181410.3390/biom12121814Advances in Gene Therapy Techniques to Treat <i>LRRK2</i> Gene MutationSun-Ku Chung0Seo-Young Lee1Division of KM Science Research, Korea Institute of Oriental Medicine, 1672 Yuseong-daero, Yuseong-gu, Daejeon 34054, Republic of KoreaDivision of KM Science Research, Korea Institute of Oriental Medicine, 1672 Yuseong-daero, Yuseong-gu, Daejeon 34054, Republic of KoreaLeucine-rich repeat kinase 2 (<i>LRRK2</i>) gene mutation is an autosomal dominant mutation associated with Parkinson’s disease (PD). Among <i>LRRK2</i> gene mutations, the <i>LRRK2</i> G2019S mutation is frequently involved in PD onset. Currently, diverse gene correction tools such as zinc finger nucleases (ZFNs), helper-dependent adenoviral vector (HDAdV), the bacterial artificial chromosome-based homologous recombination (BAC-based HR) system, and CRISPR/Cas9-homology-directed repair (HDR) or adenine base editor (ABE) are used in genome editing. Gene correction of the <i>LRRK2</i> G2019S mutation has been applied whenever new gene therapy tools emerge, being mainly applied to induced pluripotent stem cells (<i>LRRK2</i> G2019S-mutant iPSCs). Here, we comprehensively introduce the principles and methods of each programmable nuclease such as ZFN, CRISPR/Cas9-HDR or ABE applied to <i>LRRK2</i> G2019S, as well as those of HDAdV or BAC-based HR systems used as nonprogrammable nuclease systems.https://www.mdpi.com/2218-273X/12/12/1814<i>LRRK2</i> G2019Szinc finger nucleaseshelper-dependent adenoviral vectorCRISPR/Cas9-HDRadenine base editorbacterial artificial chromosome-based homologous recombination |
| spellingShingle | Sun-Ku Chung Seo-Young Lee Advances in Gene Therapy Techniques to Treat <i>LRRK2</i> Gene Mutation Biomolecules <i>LRRK2</i> G2019S zinc finger nucleases helper-dependent adenoviral vector CRISPR/Cas9-HDR adenine base editor bacterial artificial chromosome-based homologous recombination |
| title | Advances in Gene Therapy Techniques to Treat <i>LRRK2</i> Gene Mutation |
| title_full | Advances in Gene Therapy Techniques to Treat <i>LRRK2</i> Gene Mutation |
| title_fullStr | Advances in Gene Therapy Techniques to Treat <i>LRRK2</i> Gene Mutation |
| title_full_unstemmed | Advances in Gene Therapy Techniques to Treat <i>LRRK2</i> Gene Mutation |
| title_short | Advances in Gene Therapy Techniques to Treat <i>LRRK2</i> Gene Mutation |
| title_sort | advances in gene therapy techniques to treat i lrrk2 i gene mutation |
| topic | <i>LRRK2</i> G2019S zinc finger nucleases helper-dependent adenoviral vector CRISPR/Cas9-HDR adenine base editor bacterial artificial chromosome-based homologous recombination |
| url | https://www.mdpi.com/2218-273X/12/12/1814 |
| work_keys_str_mv | AT sunkuchung advancesingenetherapytechniquestotreatilrrk2igenemutation AT seoyounglee advancesingenetherapytechniquestotreatilrrk2igenemutation |