P E R S O N A L I Z E D    G E N E    T H E R A P Y
Design and development of next-generation gene therapies.

​Human gene therapy has been a topic of controversy for decades, comprising of both successes and failures. Behind this matter is an unmet need for the multitude of genetic disorders lacking absolute treatments. The past decade has witnessed the revolutionary emergence and continued refinement of numerous gene-editing tools. These include CRISPR/Cas, multiple DNA & RNA variants, viral & nano-particle based delivery mechanisms, among others. Translational and therapeutic advances have arisen from progress in both practicality and safety, with multiple clinical trials using these tools currently underway. However, there are still many barriers left to overcome. Gene therapy applications are still in their infancy, and require further research and development. STEM Biomedical strives to continue this research to fulfill the promising potential of gene therapy as a viable treatment for numerous diseases. ​
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Molecular graphics and analyses performed with UCSF Chimera, developed by the Resource for Biocomputing, Visualization, and Informatics at the University of California, San Francisco, with support from NIH P41-GM103311. UCSF Chimera--a visualization system for exploratory research and analysis. Pettersen EF, Goddard TD, Huang CC, Couch GS, Greenblatt DM, Meng EC, Ferrin TE. J Comput Chem. 2004 Oct;25(13):1605-12.