Gene Therapy Using Efficient Direct Lineage Reprogramming Technology for Neurological Diseases
Gene therapy is an innovative approach in the field of regenerative medicine. This therapy entails the transfer of genetic material into a patient’s cells to treat diseases. In particular, gene therapy for neurological diseases has recently achieved significant progress, with numerous studies invest...
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| Format: | Article |
| Language: | English |
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MDPI AG
2023-05-01
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| Series: | Nanomaterials |
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| Online Access: | https://www.mdpi.com/2079-4991/13/10/1680 |
| _version_ | 1797598842114277376 |
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| author | Yujung Chang Sungwoo Lee Jieun Kim Chunggoo Kim Hyun Soo Shim Seung Eun Lee Hyeok Ju Park Jeongwon Kim Soohyun Lee Yong Kyu Lee Sungho Park Junsang Yoo |
| author_facet | Yujung Chang Sungwoo Lee Jieun Kim Chunggoo Kim Hyun Soo Shim Seung Eun Lee Hyeok Ju Park Jeongwon Kim Soohyun Lee Yong Kyu Lee Sungho Park Junsang Yoo |
| author_sort | Yujung Chang |
| collection | DOAJ |
| description | Gene therapy is an innovative approach in the field of regenerative medicine. This therapy entails the transfer of genetic material into a patient’s cells to treat diseases. In particular, gene therapy for neurological diseases has recently achieved significant progress, with numerous studies investigating the use of adeno-associated viruses for the targeted delivery of therapeutic genetic fragments. This approach has potential applications for treating incurable diseases, including paralysis and motor impairment caused by spinal cord injury and Parkinson’s disease, and it is characterized by dopaminergic neuron degeneration. Recently, several studies have explored the potential of direct lineage reprogramming (DLR) for treating incurable diseases, and highlighted the advantages of DLR over conventional stem cell therapy. However, application of DLR technology in clinical practice is hindered by its low efficiency compared with cell therapy using stem cell differentiation. To overcome this limitation, researchers have explored various strategies such as the efficiency of DLR. In this study, we focused on innovative strategies, including the use of a nanoporous particle-based gene delivery system to improve the reprogramming efficiency of DLR-induced neurons. We believe that discussing these approaches can facilitate the development of more effective gene therapies for neurological disorders. |
| first_indexed | 2024-03-11T03:26:27Z |
| format | Article |
| id | doaj.art-65f150bd79ff4c7c9a5c473841551e8d |
| institution | Directory Open Access Journal |
| issn | 2079-4991 |
| language | English |
| last_indexed | 2024-03-11T03:26:27Z |
| publishDate | 2023-05-01 |
| publisher | MDPI AG |
| record_format | Article |
| series | Nanomaterials |
| spelling | doaj.art-65f150bd79ff4c7c9a5c473841551e8d2023-11-18T02:43:10ZengMDPI AGNanomaterials2079-49912023-05-011310168010.3390/nano13101680Gene Therapy Using Efficient Direct Lineage Reprogramming Technology for Neurological DiseasesYujung Chang0Sungwoo Lee1Jieun Kim2Chunggoo Kim3Hyun Soo Shim4Seung Eun Lee5Hyeok Ju Park6Jeongwon Kim7Soohyun Lee8Yong Kyu Lee9Sungho Park10Junsang Yoo11Laboratory of Regenerative Medicine for Neurodegenerative Disease, Stand Up Therapeutics, Hannamdaero 98, Seoul 04418, Republic of KoreaDepartment of Chemistry, Sungkyunkwan University, 2066, Seobu-ro, Jangan-gu, Suwon-si 16419, Republic of KoreaDepartment of Bio-Health Technology, College of Biomedical Science, Kangwon National University, 1 Kangwondeahak-gil, Chuncheon 24341, Republic of KoreaLaboratory of Regenerative Medicine for Neurodegenerative Disease, Stand Up Therapeutics, Hannamdaero 98, Seoul 04418, Republic of KoreaLaboratory of Regenerative Medicine for Neurodegenerative Disease, Stand Up Therapeutics, Hannamdaero 98, Seoul 04418, Republic of KoreaResearch Animal Resource Center, Korea Institute of Science and Technology, Hwarang-ro 14-gil, Seongbuk-gu, Seoul 02792, Republic of KoreaDatabase Laboratory, Department of Computer Science and Engineering, Dongguk University-Seoul, Pildong-ro 1-gil 30, Jung-gu, Seoul 04620, Republic of KoreaDepartment of Chemistry, Sungkyunkwan University, 2066, Seobu-ro, Jangan-gu, Suwon-si 16419, Republic of KoreaDepartment of Chemistry, Sungkyunkwan University, 2066, Seobu-ro, Jangan-gu, Suwon-si 16419, Republic of KoreaDatabase Laboratory, Department of Computer Science and Engineering, Dongguk University-Seoul, Pildong-ro 1-gil 30, Jung-gu, Seoul 04620, Republic of KoreaDepartment of Chemistry, Sungkyunkwan University, 2066, Seobu-ro, Jangan-gu, Suwon-si 16419, Republic of KoreaLaboratory of Regenerative Medicine for Neurodegenerative Disease, Stand Up Therapeutics, Hannamdaero 98, Seoul 04418, Republic of KoreaGene therapy is an innovative approach in the field of regenerative medicine. This therapy entails the transfer of genetic material into a patient’s cells to treat diseases. In particular, gene therapy for neurological diseases has recently achieved significant progress, with numerous studies investigating the use of adeno-associated viruses for the targeted delivery of therapeutic genetic fragments. This approach has potential applications for treating incurable diseases, including paralysis and motor impairment caused by spinal cord injury and Parkinson’s disease, and it is characterized by dopaminergic neuron degeneration. Recently, several studies have explored the potential of direct lineage reprogramming (DLR) for treating incurable diseases, and highlighted the advantages of DLR over conventional stem cell therapy. However, application of DLR technology in clinical practice is hindered by its low efficiency compared with cell therapy using stem cell differentiation. To overcome this limitation, researchers have explored various strategies such as the efficiency of DLR. In this study, we focused on innovative strategies, including the use of a nanoporous particle-based gene delivery system to improve the reprogramming efficiency of DLR-induced neurons. We believe that discussing these approaches can facilitate the development of more effective gene therapies for neurological disorders.https://www.mdpi.com/2079-4991/13/10/1680cell fate conversiondirect lineage reprogrammingspinal cord injurygene therapynanoporous particle-based gene delivery |
| spellingShingle | Yujung Chang Sungwoo Lee Jieun Kim Chunggoo Kim Hyun Soo Shim Seung Eun Lee Hyeok Ju Park Jeongwon Kim Soohyun Lee Yong Kyu Lee Sungho Park Junsang Yoo Gene Therapy Using Efficient Direct Lineage Reprogramming Technology for Neurological Diseases Nanomaterials cell fate conversion direct lineage reprogramming spinal cord injury gene therapy nanoporous particle-based gene delivery |
| title | Gene Therapy Using Efficient Direct Lineage Reprogramming Technology for Neurological Diseases |
| title_full | Gene Therapy Using Efficient Direct Lineage Reprogramming Technology for Neurological Diseases |
| title_fullStr | Gene Therapy Using Efficient Direct Lineage Reprogramming Technology for Neurological Diseases |
| title_full_unstemmed | Gene Therapy Using Efficient Direct Lineage Reprogramming Technology for Neurological Diseases |
| title_short | Gene Therapy Using Efficient Direct Lineage Reprogramming Technology for Neurological Diseases |
| title_sort | gene therapy using efficient direct lineage reprogramming technology for neurological diseases |
| topic | cell fate conversion direct lineage reprogramming spinal cord injury gene therapy nanoporous particle-based gene delivery |
| url | https://www.mdpi.com/2079-4991/13/10/1680 |
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