Therapeutics in paediatric genetic diseases: current and future landscape
There are more than 7,000 paediatric genetic diseases (PGDs) but less than 5% have treatment options. Treatment strategies targeting different levels of the biological process of the disease have led to optimal health outcomes in a subset of patients with PGDs, where treatment is available. In the p...
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| Format: | Article |
| Language: | English |
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Wolters Kluwer – Medknow Publications
2023-01-01
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| Series: | Singapore Medical Journal |
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| Online Access: | https://journals.lww.com/10.4103/singaporemedj.SMJ-2021-376 |
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| _version_ | 1823861448961425408 |
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| author | Ai Ling Koh Saumya Shekhar Jamuar |
| author_facet | Ai Ling Koh Saumya Shekhar Jamuar |
| author_sort | Ai Ling Koh |
| collection | DOAJ |
| description | There are more than 7,000 paediatric genetic diseases (PGDs) but less than 5% have treatment options. Treatment strategies targeting different levels of the biological process of the disease have led to optimal health outcomes in a subset of patients with PGDs, where treatment is available. In the past 3 decades, there has been rapid advancement in the development of novel therapies, including gene therapy, for many PGDs. The therapeutic success of treatment relies heavily on knowledge of the genetic basis and the disease mechanism. Specifically, gene therapy has been shown to be effective in various clinical trials, and indeed, these trials have led to regulatory approvals, paving the way for gene therapies for other types of PGDs. In this review, we provide an overview of the treatment strategies and focus on some of the recent advancements in therapeutics for PGDs. |
| format | Article |
| id | doaj-art-1a7521d0ae8b4f9aa8aa8d057a4fb791 |
| institution | Kabale University |
| issn | 0037-5675 2737-5935 |
| language | English |
| publishDate | 2023-01-01 |
| publisher | Wolters Kluwer – Medknow Publications |
| record_format | Article |
| series | Singapore Medical Journal |
| spelling | doaj-art-1a7521d0ae8b4f9aa8aa8d057a4fb7912025-02-09T13:24:03ZengWolters Kluwer – Medknow PublicationsSingapore Medical Journal0037-56752737-59352023-01-0164171610.4103/singaporemedj.SMJ-2021-376Therapeutics in paediatric genetic diseases: current and future landscapeAi Ling KohSaumya Shekhar JamuarThere are more than 7,000 paediatric genetic diseases (PGDs) but less than 5% have treatment options. Treatment strategies targeting different levels of the biological process of the disease have led to optimal health outcomes in a subset of patients with PGDs, where treatment is available. In the past 3 decades, there has been rapid advancement in the development of novel therapies, including gene therapy, for many PGDs. The therapeutic success of treatment relies heavily on knowledge of the genetic basis and the disease mechanism. Specifically, gene therapy has been shown to be effective in various clinical trials, and indeed, these trials have led to regulatory approvals, paving the way for gene therapies for other types of PGDs. In this review, we provide an overview of the treatment strategies and focus on some of the recent advancements in therapeutics for PGDs.https://journals.lww.com/10.4103/singaporemedj.SMJ-2021-376antisense oligonucleotidegene therapypaediatric genetic diseasestherapeutics |
| spellingShingle | Ai Ling Koh Saumya Shekhar Jamuar Therapeutics in paediatric genetic diseases: current and future landscape Singapore Medical Journal antisense oligonucleotide gene therapy paediatric genetic diseases therapeutics |
| title | Therapeutics in paediatric genetic diseases: current and future landscape |
| title_full | Therapeutics in paediatric genetic diseases: current and future landscape |
| title_fullStr | Therapeutics in paediatric genetic diseases: current and future landscape |
| title_full_unstemmed | Therapeutics in paediatric genetic diseases: current and future landscape |
| title_short | Therapeutics in paediatric genetic diseases: current and future landscape |
| title_sort | therapeutics in paediatric genetic diseases current and future landscape |
| topic | antisense oligonucleotide gene therapy paediatric genetic diseases therapeutics |
| url | https://journals.lww.com/10.4103/singaporemedj.SMJ-2021-376 |
| work_keys_str_mv | AT ailingkoh therapeuticsinpaediatricgeneticdiseasescurrentandfuturelandscape AT saumyashekharjamuar therapeuticsinpaediatricgeneticdiseasescurrentandfuturelandscape |