Significance of gene therapy in neurodegenerative diseases
Gene therapy is an approach that employs vectors to deliver genetic material to target cells, aiming to correct genes with pathogenic mutations and modulate one or more genes responsible for disease progression. It holds significant value for clinical applications and offers broad market potential d...
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| Format: | Article |
| Language: | English |
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Frontiers Media S.A.
2025-05-01
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| Series: | Frontiers in Neuroscience |
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| Online Access: | https://www.frontiersin.org/articles/10.3389/fnins.2025.1515255/full |
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| author | Lingling Wang Lin Ma Zihan Gao Ying Wang Jiaoxue Qiu |
| author_facet | Lingling Wang Lin Ma Zihan Gao Ying Wang Jiaoxue Qiu |
| author_sort | Lingling Wang |
| collection | DOAJ |
| description | Gene therapy is an approach that employs vectors to deliver genetic material to target cells, aiming to correct genes with pathogenic mutations and modulate one or more genes responsible for disease progression. It holds significant value for clinical applications and offers broad market potential due to the large patient population affected by various conditions. For instance, in 2023, the Food and Drug Administration (FDA) approved 55 new drugs, including five specifically for gene therapy targeting hematologic and rare diseases. Recently, with advancements in understanding the pathogenesis and development of neurodegenerative diseases (NDDs), gene therapy has emerged as a promising avenue for treating Alzheimer's disease (AD), Parkinson's disease (PD), Huntington's disease (HD), amyotrophic lateral sclerosis (ALS), and spinal muscular atrophy (SMA), particularly in personalized medicine. Notably, the FDA has approved three clinical applications for combating SMA, utilizing viral vectors delivered via intravenous and intrathecal injections. However, gene therapy for other NDDs remains in clinical trials, necessitating improvements in viral vectors, exploration of new vectors, optimization of delivery routes, and further investigation into pathogenesis to identify novel targets. This review discusses recent advancements in gene therapy for NDDs, offering insights into developing new therapeutic strategies. |
| format | Article |
| id | doaj-art-0369292420894cd4b0145a84b0d01a9e |
| institution | OA Journals |
| issn | 1662-453X |
| language | English |
| publishDate | 2025-05-01 |
| publisher | Frontiers Media S.A. |
| record_format | Article |
| series | Frontiers in Neuroscience |
| spelling | doaj-art-0369292420894cd4b0145a84b0d01a9e2025-08-20T02:27:06ZengFrontiers Media S.A.Frontiers in Neuroscience1662-453X2025-05-011910.3389/fnins.2025.15152551515255Significance of gene therapy in neurodegenerative diseasesLingling Wang0Lin Ma1Zihan Gao2Ying Wang3Jiaoxue Qiu4Department of Neurology, Yantai Shan Hospital, Yantai, ChinaDepartment of Neurology, Qingdao Municipal Hospital, Qingdao, ChinaDepartment of Internal Medicine of Traditional Chinese Medicine, Affiliated Hospital of Shandong University of Traditional Chinese Medicine, Jinan, ChinaDepartment of Neurology, Yantai Shan Hospital, Yantai, ChinaDepartment of Neurology, Yantai Shan Hospital, Yantai, ChinaGene therapy is an approach that employs vectors to deliver genetic material to target cells, aiming to correct genes with pathogenic mutations and modulate one or more genes responsible for disease progression. It holds significant value for clinical applications and offers broad market potential due to the large patient population affected by various conditions. For instance, in 2023, the Food and Drug Administration (FDA) approved 55 new drugs, including five specifically for gene therapy targeting hematologic and rare diseases. Recently, with advancements in understanding the pathogenesis and development of neurodegenerative diseases (NDDs), gene therapy has emerged as a promising avenue for treating Alzheimer's disease (AD), Parkinson's disease (PD), Huntington's disease (HD), amyotrophic lateral sclerosis (ALS), and spinal muscular atrophy (SMA), particularly in personalized medicine. Notably, the FDA has approved three clinical applications for combating SMA, utilizing viral vectors delivered via intravenous and intrathecal injections. However, gene therapy for other NDDs remains in clinical trials, necessitating improvements in viral vectors, exploration of new vectors, optimization of delivery routes, and further investigation into pathogenesis to identify novel targets. This review discusses recent advancements in gene therapy for NDDs, offering insights into developing new therapeutic strategies.https://www.frontiersin.org/articles/10.3389/fnins.2025.1515255/fullgene therapyAlzheimer's disease (AD)Parkinson's disease (PD)Huntington disease (HD)amyotrophic lateral sclerosis (ALS)spinal muscular atrophy (SMA) |
| spellingShingle | Lingling Wang Lin Ma Zihan Gao Ying Wang Jiaoxue Qiu Significance of gene therapy in neurodegenerative diseases Frontiers in Neuroscience gene therapy Alzheimer's disease (AD) Parkinson's disease (PD) Huntington disease (HD) amyotrophic lateral sclerosis (ALS) spinal muscular atrophy (SMA) |
| title | Significance of gene therapy in neurodegenerative diseases |
| title_full | Significance of gene therapy in neurodegenerative diseases |
| title_fullStr | Significance of gene therapy in neurodegenerative diseases |
| title_full_unstemmed | Significance of gene therapy in neurodegenerative diseases |
| title_short | Significance of gene therapy in neurodegenerative diseases |
| title_sort | significance of gene therapy in neurodegenerative diseases |
| topic | gene therapy Alzheimer's disease (AD) Parkinson's disease (PD) Huntington disease (HD) amyotrophic lateral sclerosis (ALS) spinal muscular atrophy (SMA) |
| url | https://www.frontiersin.org/articles/10.3389/fnins.2025.1515255/full |
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