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...
Saved in:
| Main Authors: | Ai Ling Koh, Saumya Shekhar Jamuar |
|---|---|
| Format: | Article |
| Language: | English |
| Published: |
Wolters Kluwer – Medknow Publications
2023-01-01
|
| Series: | Singapore Medical Journal |
| Subjects: | |
| Online Access: | https://journals.lww.com/10.4103/singaporemedj.SMJ-2021-376 |
| Tags: |
Add Tag
No Tags, Be the first to tag this record!
|
Similar Items
-
Nucleic acid therapeutics: Past, present, and future
by: Sajid Naeem, et al.
Published: (2025-03-01) -
Design and screening of novel endosomal escape compounds that enhance functional delivery of oligonucleotides in vitro
by: H. Yesid Estupiñán, et al.
Published: (2025-06-01) -
ANTISENSE OLIGONUCLEOTIDES AND THERAPEUTICAL MONOCLONAL ANTIBODIES AS A BASEMENT FOR NOVEL BIOLOGICAL LIPIDLOWERING DRUGS
by: O. I. Afanasieva, et al.
Published: (2018-09-01) -
Therapeutic correction of ApoER2 splicing in Alzheimer's disease mice using antisense oligonucleotides
by: Anthony J Hinrich, et al.
Published: (2016-02-01) -
Multispanning membrane protein SIDT2 increases knockdown activity of gapmer antisense oligonucleotides
by: Kohshi Kusumoto, et al.
Published: (2025-01-01)