Pharmacologic target gene modulation is the primary objective for RNA antagonist strategies and gene therapy. Here we show that mRNAs encoding tissue-specific gene transcripts can be detected in biological fluids and that RNAi-mediated target gene silencing in the liver and brain results in quantitative reductions in serum and cerebrospinal fluid mRNA levels, respectively. Further, administrati...

RNA silencing is an established method for investigating gene function and has attracted particular interest because of the potential for generating RNA-based therapeutics. Using lentiviral vectors as an efficient delivery system that offers stable, long-term expression in postmitotic cells further enhances the applicability of an RNA-based gene therapy for the CNS. In this review we provide an...

Abstract BACKGROUND Glioblastoma multiforme (GBM) remains a disease with debilitating survival outcomes. Cancer immunogenomics represents complementary approach to the application of genomics in developing novel immunotherapies for brain malignancies. Using cancer that we developed, called Open Reading Frame Antigen Network (O.R.A.N.), identified immunogenic neoantigens and tumor-associated ant...

Combinatorial therapies for the treatment of HIV-1 infection have proven to be effective in reducing patient viral loads and slowing the progression to AIDS. We have developed a series of RNA-based inhibitors for use in a gene therapy-based treatment for HIV-1 infection. The transcriptional units have been inserted into the backbone of a replication-defective lentiviral vector capable of transd...

Introduction : The process of protein synthesis is a vital for all kingdoms life. ribosome ribonucleoprotein complex that reads the genetic code, from messenger RNA (mRNA) to produce proteins and tightly regulate ensure cells growth. fact numerous diseases are caused by defect during biogenesis important understand this pathway. Materials methods We have analyzed literature its links with diffe...

background: rna plays key role in many aspects of biological processes and its tertiary structure is critical for its biological function. rna secondary structure represents various significant portions of rna tertiary structure. since the biological function of rna is concluded indirectly from its primary structure, it would be important to analyze the relations between the rna sequences and t...

Biologic drugs have revolutionised outcomes in psoriasis, with a growing number of patients achieving clear skin (‘remission’). However, treatment involves expensive regular injections, and the long-term impact remains uncertain. An improved understanding early mechanisms underpinning biologics-induced remission may help inform strategies around drug withdrawal. This promises to overcome burden...