The potential of antisense oligonucleotide therapies for inherited childhood lung diseases.
Antisense oligonucleotides are an emerging therapeutic option to treat diseases with known genetic origin. In the age of personalised medicines, antisense oligonucleotides can sometimes be designed to target and bypass or overcome a patient's genetic mutation, in particular those lesions that c...
| Main Authors: | , , , , , , |
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| Format: | Journal Article |
| Language: | English |
| Published: |
2018
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| Subjects: | |
| Online Access: | http://hdl.handle.net/20.500.11937/76807 |
| _version_ | 1848763765042970624 |
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| author | Martinovich, K. Shaw, N. Kicic, Anthony Schultz, A. Fletcher, S. Wilton, S. Stick, S. |
| author_facet | Martinovich, K. Shaw, N. Kicic, Anthony Schultz, A. Fletcher, S. Wilton, S. Stick, S. |
| author_sort | Martinovich, K. |
| building | Curtin Institutional Repository |
| collection | Online Access |
| description | Antisense oligonucleotides are an emerging therapeutic option to treat diseases with known genetic origin. In the age of personalised medicines, antisense oligonucleotides can sometimes be designed to target and bypass or overcome a patient's genetic mutation, in particular those lesions that compromise normal pre-mRNA processing. Antisense oligonucleotides can alter gene expression through a variety of mechanisms as determined by the chemistry and antisense oligomer design. Through targeting the pre-mRNA, antisense oligonucleotides can alter splicing and induce a specific spliceoform or disrupt the reading frame, target an RNA transcript for degradation through RNaseH activation, block ribosome initiation of protein translation or disrupt miRNA function. The recent accelerated approval of eteplirsen (renamed Exondys 51™) by the Food and Drug Administration, for the treatment of Duchenne muscular dystrophy, and nusinersen, for the treatment of spinal muscular atrophy, herald a new and exciting era in splice-switching antisense oligonucleotide applications to treat inherited diseases. This review considers the potential of antisense oligonucleotides to treat inherited lung diseases of childhood with a focus on cystic fibrosis and disorders of surfactant protein metabolism. |
| first_indexed | 2025-11-14T11:08:39Z |
| format | Journal Article |
| id | curtin-20.500.11937-76807 |
| institution | Curtin University Malaysia |
| institution_category | Local University |
| language | eng |
| last_indexed | 2025-11-14T11:08:39Z |
| publishDate | 2018 |
| recordtype | eprints |
| repository_type | Digital Repository |
| spelling | curtin-20.500.11937-768072019-11-11T06:50:43Z The potential of antisense oligonucleotide therapies for inherited childhood lung diseases. Martinovich, K. Shaw, N. Kicic, Anthony Schultz, A. Fletcher, S. Wilton, S. Stick, S. Antisense oligonucleotides Childhood Cystic fibrosis Inherited diseases Surfactant disorders Antisense oligonucleotides are an emerging therapeutic option to treat diseases with known genetic origin. In the age of personalised medicines, antisense oligonucleotides can sometimes be designed to target and bypass or overcome a patient's genetic mutation, in particular those lesions that compromise normal pre-mRNA processing. Antisense oligonucleotides can alter gene expression through a variety of mechanisms as determined by the chemistry and antisense oligomer design. Through targeting the pre-mRNA, antisense oligonucleotides can alter splicing and induce a specific spliceoform or disrupt the reading frame, target an RNA transcript for degradation through RNaseH activation, block ribosome initiation of protein translation or disrupt miRNA function. The recent accelerated approval of eteplirsen (renamed Exondys 51™) by the Food and Drug Administration, for the treatment of Duchenne muscular dystrophy, and nusinersen, for the treatment of spinal muscular atrophy, herald a new and exciting era in splice-switching antisense oligonucleotide applications to treat inherited diseases. This review considers the potential of antisense oligonucleotides to treat inherited lung diseases of childhood with a focus on cystic fibrosis and disorders of surfactant protein metabolism. 2018 Journal Article http://hdl.handle.net/20.500.11937/76807 10.1186/s40348-018-0081-6 eng http://creativecommons.org/licenses/by/4.0/ fulltext |
| spellingShingle | Antisense oligonucleotides Childhood Cystic fibrosis Inherited diseases Surfactant disorders Martinovich, K. Shaw, N. Kicic, Anthony Schultz, A. Fletcher, S. Wilton, S. Stick, S. The potential of antisense oligonucleotide therapies for inherited childhood lung diseases. |
| title | The potential of antisense oligonucleotide therapies for inherited childhood lung diseases. |
| title_full | The potential of antisense oligonucleotide therapies for inherited childhood lung diseases. |
| title_fullStr | The potential of antisense oligonucleotide therapies for inherited childhood lung diseases. |
| title_full_unstemmed | The potential of antisense oligonucleotide therapies for inherited childhood lung diseases. |
| title_short | The potential of antisense oligonucleotide therapies for inherited childhood lung diseases. |
| title_sort | potential of antisense oligonucleotide therapies for inherited childhood lung diseases. |
| topic | Antisense oligonucleotides Childhood Cystic fibrosis Inherited diseases Surfactant disorders |
| url | http://hdl.handle.net/20.500.11937/76807 |