The TRND Program initiated a suite of collaborations with biotech and academic groups that were strategically selected to serve as inaugural pilot projects. The overall goal is to enable TRND to build a gene therapy translation toolbox. New technologies to scale up gene vector manufacturing and to deliver the transgene to the right tissue at the right time and dosage are among those being developed at TRND. These technologies, along with best practices to achieve regulatory approval of gene therapy, will help improve the speed of development and reduce costs for gene therapy in general. The pilot projects include preclinical development of therapies for Duchenne muscular dystrophy, Pompe disease, and aromatic L-amino acid decarboxylase (AADC) deficiency.

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Kodippili, Kasun; Hakim, Chady H; Pan, Xiufang et al. (2018) Dual AAV Gene Therapy for Duchenne Muscular Dystrophy with a 7-kb Mini-Dystrophin Gene in the Canine Model. Hum Gene Ther 29:299-311
Nance, Michael E; Hakim, Chady H; Yang, N Nora et al. (2018) Nanotherapy for Duchenne muscular dystrophy. Wiley Interdiscip Rev Nanomed Nanobiotechnol 10:
Brooks, Philip J; Yang, N Nora; Austin, Christopher P (2016) Gene Therapy: The View from NCATS. Hum Gene Ther 27:7-13
Zhao, Junling; Kodippili, Kasun; Yue, Yongping et al. (2016) Dystrophin contains multiple independent membrane-binding domains. Hum Mol Genet 25:3647-3653