Abstract
Scientific research in recent years has made every endeavor to combat against various untreatable diseases in neurodegenerative or genetic diseases under the guidance of constantly updated underlying gene-associated pathological mechanisms. However, substantial therapeutic challenges still remain and many original causes of diseases are under great debate. Problems of drugs for neurological disorders involve high resistance to drug molecules by the blood-brain barrier (BBB) and only work efficiently in certain populations of patients, which is also a side effect of genetic malady. The introduction of Antisense oligonucleotides (ASOs) brings a new era for these diseases and meets the requirement. The US Food and Drug Administration (FDA) has proven ten oligonucleotide drugs until 2020. This article reviews some of their clinical trials with mechanisms, up-to-date attempts in inventing new ASO therapy, continuous safety and efficacy in preclinical and clinical trials of proven ASO, and great potential usage for RNA virus therapy.
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