miRNA Replacement Therapy

For more information: http://www.mirnarx.com/___Research/miRNATherapy.aspx

In normal cells, a given tumor suppressor miRNA is expressed at a certain level that is required and sufficient in modulating gene expression to maintain a normal state of its associated pathways. Due to genetic aberrations, which are part of the cancer process, this miRNA might be expressed at a reduced level which results in increased levels of many mRNAs and consequently, increased activity of their associated pathways. Since many of these genes originally suppressed by the miRNA encode key oncogenes, the loss of the tumor suppressor miRNA may lead to a conversion to the cancerous state. siRNA therapeutic approaches take advantage of the RNAi pathway and are designed to target a single gene of interest and to induce complete degradation of the target. The rationale for choosing a particular siRNA target is based on scientifically sound projections of whether inhibition might provide a therapeutic benefit. The successful eradication of the intended target leads to an inhibition of the associated pathway.

Mirna's miRNA Replacement Therapy seeks to replenish the missing tumor suppressor miRNA by introducing a mimic of the tumor suppressor miRNA. This miRNA mimic functions similarly to the endogenous miRNA and modulates the entire spectrum of genes and pathways that is also controlled in normal cells by the endogenous miRNA. Thus, tumor suppressor miRNA mimics provide an opportunity to target multiple cancer-relevant genes in a fine-tuned manner. Considerations of "off-target" effects and the selection of the "best target" become irrelevant as miRNA mimics behave like the natural counterparts for which the proper miRNA-target interactions have evolved over a billion years.

Category:

Science & Technology

Tags:

• miRNA
• microRNA
• miRNA replacement
• microRNA replacement