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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 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.

Citation:
Martinovich KM, Shaw NC, Kicic A, Schultz A, Fletcher S, Wilton SD, Stick SM. The potential of antisense oligonucleotide therapies for inherited childhood lung diseases. Mol Cell Pediatr. 2018;5(1).

Keywords:
Antisense oligonucleotides; Childhood; Cystic fibrosis; Inherited diseases; Surfactant disorders

Abstract:
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.