Cell-Based Assay Type 1

---

•  Cell-Based Assay of rAAV Potency

One of the main challenges in the gene therapy viral vector development is to establish an optimized process for its large-scale production. Between upstream rAAV design/vector construction/virus product and downstream large-scale manufacture, step-by-step analytical characterization of the AAV vector and demonstration of safety and potency of AAV vector are especially pivotal, the results of which inform the iterative refinement of production for yield, purity and potency. Beyond Biotech employ cell-based assay to demonstrate safety and potency of your AAV vectors, which provide more secure to your rAAV manufacture for current Good Manufacturing Processes (cGMP).

• Cell-Based Assay of Nanoparticle Carriers

Various formulation of nanoparticles has been used for vector delivery of gene therapy, which have benefits to targeting on site of action and reduction of toxicity/side effects. Due to the development of nano technology, a number of nanoparticles are optimized and used for the gene delivery based on the different chemical carriers, such as polymeric nanoparticles, lipid nanoparticles, magnetic nanoparticles, mesoporous silica nanoparticles, etc. Here, Beyond Biotech employ cell-based assay to screen the best nanoparticle formulation for your specific vectors of gene therapy in the specific cell populations. 

• Cell-Based Assay for gene expression or shRNA/miRNA vectors knockdown efficiency

It is especially important that target genes can be expressed in the cells after transfection. Beyond Biotech employ cell-based assay to check your vectors integrity, both mRNA and protein levels.

Cell-based assay of rAAV based shRNA/miRNA vectors knockdown efficiency short hairpin RNA (shRNA) and Artificial microRNA (miRNA) are shown to result in specific, efficient, and stable knockdown of various targeted endo- or exogenous genes in cultured mammalian cells and in animal models. Currently, rapid development and commercialization of gene therapy made a great progress due to approval of several rAAV products by FDA, which AAV vectors as vehicles for safe and efficient gene delivery. Therefore, rAAV based shRNA and artificial miRNA knockdown vector system can be an efficient method for stable knocking down expression of a target gene both in vitro and in vivo.