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dc.contributor.authorMartinovich, K.
dc.contributor.authorShaw, N.
dc.contributor.authorKicic, Anthony
dc.contributor.authorSchultz, A.
dc.contributor.authorFletcher, S.
dc.contributor.authorWilton, S.
dc.contributor.authorStick, S.
dc.identifier.citationMartinovich, K.M. and Shaw, N.C. and Kicic, A. and Schultz, A. and Fletcher, S. and Wilton, S.D. and Stick, S.M. 2018. The potential of antisense oligonucleotide therapies for inherited childhood lung diseases. Molecular and Cellular Pediatrics. 5: 3.

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.

dc.subjectAntisense oligonucleotides
dc.subjectCystic fibrosis
dc.subjectInherited diseases
dc.subjectSurfactant disorders
dc.titleThe potential of antisense oligonucleotide therapies for inherited childhood lung diseases.
dc.typeJournal Article
dcterms.source.titleMolecular and Cellular Pediatrics
curtin.departmentSchool of Public Health
curtin.accessStatusOpen access
curtin.facultyFaculty of Health Sciences
curtin.contributor.orcidKicic, Anthony [0000-0002-0008-9733]
curtin.contributor.scopusauthoridKicic, Anthony [6507472922]

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