Delivery of Genome Engineering Technologies for Precision Gene Therapy

Date(s) - 02/06/2017
3:00 pm

Christopher Nelson, Ph.D., Postdoctoral Scholar, Department of Biomedical Engineering, Duke University


Recent advances in genome engineering have enabled the precise control over gene sequence and expression. These developments have unlocked exciting potential for precision medicine including the generation of new animal models of disease and engineering of cell and gene therapies. The grand challenge for in vivo genome engineering is the development of safe and effective delivery vehicles. In this talk, I will present one example of addressing this challenge to correct the genetic basis of Duchenne muscular dystrophy (DMD). We used adeno associated virus to deliver the components of the CRISPR/Cas9 system and repair the genetic mutation in a mouse model of DMD. Gene editing restored dystrophin protein in skeletal and cardiac muscle, and led to improvements in muscle biochemistry and skeletal muscle function. I will also discuss the development of biologically inspired gene delivery platforms for controlling gene expression and their potential role in the delivery of gene editing technologies. I will conclude by discussing potential future areas of opportunity in this field, including therapeutic gene correction for monogenetic disorders and platforms for studying genetic mechanisms of complex disorders.


Dr. Nelson completed his bachelor’s degree in biological engineering at the University of Arkansas, and his PhD from Vanderbilt University. He is currently at Duke University supported by The Hartwell Foundation Postdoctoral Fellowship.

Nelson’s primary research interests are in developing new technologies for therapeutic genome engineering. Previously, he has developed biomaterial-based platforms for drug and gene delivery including a nanoparticle for systemic siRNA administration (ACS Nano 2013) and a multifunctional scaffold for local gene silencing for regenerative medicine (Advanced Materials 2014). More recently, he has applied a genome engineering approach to treat the genetic basis of Duchenne muscular dystrophy in vivo (Science 2016). He now plans to apply gene and drug delivery to genome engineering to create precision molecular therapies, study regenerative medicine, and interrogate gene function and regulation.