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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Main Authors: Koh, Ai Ling, Jamuar, Saumya Shekhar
Other Authors: Lee Kong Chian School of Medicine (LKCMedicine)
Format: Article
Language:English
Published: 2023
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Online Access:https://hdl.handle.net/10356/169411
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Institution: Nanyang Technological University
Language: English
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spelling sg-ntu-dr.10356-1694112023-07-23T15:37:58Z Therapeutics in paediatric genetic diseases: current and future landscape Koh, Ai Ling Jamuar, Saumya Shekhar Lee Kong Chian School of Medicine (LKCMedicine) KK Women’s and Children’s Hospital SingHealth Duke‑NUS Genomic Medicine Centre Duke‑NUS Medical School Yong Loo Lin School of Medicine, NUS Science::Medicine Antisense Oligonucleotide Gene Therapy 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. Published version 2023-07-18T02:16:29Z 2023-07-18T02:16:29Z 2023 Journal Article Koh, A. L. & Jamuar, S. S. (2023). Therapeutics in paediatric genetic diseases: current and future landscape. Singapore Medical Journal, 64(1), 7-16. https://dx.doi.org/10.4103/singaporemedj.SMJ-2021-376 0037-5675 https://hdl.handle.net/10356/169411 10.4103/singaporemedj.SMJ-2021-376 36722512 2-s2.0-85147186921 1 64 7 16 en Singapore Medical Journal © 2023 Singapore Medical Journal. Published by Wolters Kluwer - Medknow. This is an open access journal, and articles are distributed under the terms of the Creative Commons Attribution‑NonCommercial‑ShareAlike 4.0 License, which allows others to remix, tweak, and build upon the work non‑commercially, as long as appropriate credit is given and the new creations are licensed under the identical terms. application/pdf
institution Nanyang Technological University
building NTU Library
continent Asia
country Singapore
Singapore
content_provider NTU Library
collection DR-NTU
language English
topic Science::Medicine
Antisense Oligonucleotide
Gene Therapy
spellingShingle Science::Medicine
Antisense Oligonucleotide
Gene Therapy
Koh, Ai Ling
Jamuar, Saumya Shekhar
Therapeutics in paediatric genetic diseases: current and future landscape
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.
author2 Lee Kong Chian School of Medicine (LKCMedicine)
author_facet Lee Kong Chian School of Medicine (LKCMedicine)
Koh, Ai Ling
Jamuar, Saumya Shekhar
format Article
author Koh, Ai Ling
Jamuar, Saumya Shekhar
author_sort Koh, Ai Ling
title Therapeutics in paediatric genetic diseases: current and future landscape
title_short 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_sort therapeutics in paediatric genetic diseases: current and future landscape
publishDate 2023
url https://hdl.handle.net/10356/169411
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