ISIS-388626
ISIS-388626
ISIS-388626 is an antisense oligonucleotide (ASO) designed to target and reduce the expression of a specific gene involved in metabolic processes. This compound is part of a broader class of therapeutic agents known as antisense drugs, which are used to modulate gene expression by binding to messenger RNA (mRNA) and preventing the production of specific proteins.
Mechanism of Action[edit | edit source]
ISIS-388626 works by binding to the mRNA of its target gene, thereby preventing the translation of the mRNA into protein. This process is known as "antisense inhibition." By reducing the levels of the target protein, ISIS-388626 can alter the physiological processes in which the protein is involved, potentially leading to therapeutic benefits.
Therapeutic Applications[edit | edit source]
ISIS-388626 has been investigated for its potential use in treating metabolic disorders, particularly those related to lipid metabolism. By modulating the expression of genes involved in these pathways, ISIS-388626 may help in managing conditions such as hyperlipidemia and other related disorders.
Clinical Trials[edit | edit source]
Clinical trials are essential for determining the safety and efficacy of new therapeutic agents like ISIS-388626. These trials typically progress through several phases, starting with Phase I trials to assess safety, followed by Phase II and III trials to evaluate efficacy and further safety in larger populations.
Side Effects and Safety[edit | edit source]
As with any therapeutic agent, the use of ISIS-388626 may be associated with side effects. Common side effects observed in clinical trials of antisense oligonucleotides include injection site reactions, flu-like symptoms, and changes in liver enzyme levels. The safety profile of ISIS-388626 is continually assessed through ongoing clinical research.
Research and Development[edit | edit source]
ISIS-388626 is developed by Ionis Pharmaceuticals, a leader in the field of antisense technology. The development of antisense drugs like ISIS-388626 represents a significant advancement in precision medicine, allowing for targeted intervention at the genetic level.
Also see[edit | edit source]
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