The Growing Potential for Antisense Oligonucleotide Therapy
Antisense oligonucleotide therapy represents a burgeoning field in therapeutics, offering a novel avenue for targeting diseases at the RNA level.
Unlike conventional therapies that focus on downstream processes, antisense oligonucleotides (ASOs) bind specifically to target RNA sequences and modulate protein expression through various mechanisms.
ASOs are composed of short, synthetic, single-stranded oligonucleotides which can modify RNA – meaning they can aim to correct the underlying pathogenesis present in chronic diseases that arise from errant protein expression.
By addressing aberrant protein production associated with diseases such as Crohn’s and amyotrophic lateral sclerosis, ASOs could provide a promising alternative approach to treatment.
However, the translation of ASO-based therapies into clinical successes hinges on overcoming challenges that may arise – these include off-target side effects and insufficient biological activity.
ASO-based therapies work through the manipulation of messenger RNA (mRNA) to regulate protein expression.
This manipulation has come with the discovery of non-coding RNAs (ncRNAs), facilitating a strategy of targeting pre-mRNA, mRNA, or ncRNAs.
The growing significance of ASOs in the broader therapeutic landscape is evidenced by recent FDA approvals for drugs based on nucleic acids.
Markedly, ASO-based therapies have gained FDA approval for Duchenne muscular dystrophy and spinal muscular atrophy, underlining their therapeutic potential.
Potential Applications of Antisense Oligonucleotide Therapy
The potential applications of ASOs could address a range of different chronic diseases which stem from errant protein expression.
Sufferers of inflammatory bowel disease, amyotrophic lateral sclerosis, Duchenne muscular dystrophy, and spinal muscular atrophy could all benefit from ASO-based treatments in years to come.
Out of the major players in pharmaceuticals, AstraZeneca has been the most enthusiastic in throwing its weight behind ASOs.
At present, the pharmaceutical developer is focusing on furthering its precision medicine approaches in NASH – a leading cause of liver disease.
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Their research focuses on how ASOs could mimic the protective effects of genetic mutations that protect against NASH and fatty liver disease.
Recent developments in human genome sequencing have potentially opened up the possibility for using ASOs to function as therapeutic agents or tools for gene function assessment.
The application of ASOs in inflammatory bowel diseases like Crohn's disease and ulcerative colitis highlights their potential to downregulate molecular targets, presenting a promising avenue for effective therapeutic strategies.
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