Gene therapy is an experimental technique that uses genes to treat or prevent disease. In the future, this technique may allow treating a disorder by inserting a gene into a patient’s cells instead of using drugs or surgery. There are numerous ongoing researches of which the focus is testing several approaches to gene therapy. Although gene therapy is a promising treatment scenario for a number of diseases, including inherited disorders, some types of cancer, and certain viral infections, there is still a number of obstacles to be overcome in terms of the therapeutic modality selection and design at the very early stage of development, design and application of in vitro and in vivo assays for proof-of-concept studies, comprehensive development of full set of assays for measuring potency and safety, from which the information should be clarified to support an Investigational New Drug Application (IND) or a Biologics License Application (BLA). 	BEST1 pH1 shRNA	https://www.creative-biolabs.com/gene-therapy/raav-2-ph1-best1-shrna-seq1-736.htm