Self-assembled Modular Platform Proposed for Targeted Therapeutics

The striking complexity of various cancer types, along with notable genotypic and phenotypic heterogeneity, calls for novel approaches in precision medicine. Such personalized therapeutics seeks to expand the current armamentarium against cancer and a range of autoimmune diseases. In a recent study at Tel Aviv University (TAUs), researchers have hit upon an innovative approach to target siRNAs by developing self-assembling modular platform for gene manipulation. The vast therapeutic platform, the authors opine, will apply to infinitely large number of disease conditions and is likely to open new vistas in precision medicine in the coming years.

The research is conducted by authors from across various institutes at the University and is led by Professor Dan Peer of the Laboratory of Precision Nanomedicine, TAU. The study is published online in the journal Nature Nanotechnology on January 29, 2018.

Platform has Potential to Target Large Number of Cellular Receptor

Current oncology practices mostly rely on targeting selected disease-related genes and pathways. Such approaches fall flat on effective cure and care for patient population world over. Hence, scientists have been relentlessly looking to expand the repertoire of targeted carriers, notably short interfering RNAs (siRNAs), mainly by manipulating gene expression and have had their share of success. However, the effective clinical translation of such therapeutics still poses formidable challenges.

Novel Approach Solves Several Limitation in Current Precision Medicine in Oncology

According to the lead author of the study, the platform hopes to solve several limitations associated with the current precision medicine in oncology. The researchers demonstrated the therapeutic efficacy of the proposed platform in a mouse model targeting an inflammatory bowel disease (IBD). The platform uses a membrane-anchored lipoprotein acting as a linker and the approach is based on affinity interactions, wherein linkers in nanoparticle membrane are bound to specific region of an antibody. Since any antibody belonging to the same isotype shares the same region and altering it induces the delivery of a large number of carriers to target cell surface receptors of choice.

The researchers are now focused on developing a simple nucleic acid-based tool that can manipulate gene expression to develop common drug carriers for targeting a wide variety of cell types. Such platform will prove promising for endless number of receptors for each patient.

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