Non-Viral Delivery for Gene Therapy and Editing

Timeslot: Friday, April 5, 2019 - 10:30am to 12:30pm
Track: Therapeutic Delivery
Room: Skagit 2

About

Targeted genome editing using programmable nucleases has recently rapidly transformed from a technique on the bench to a potential avenue for the treatment of genetic disorders and diseases. Three main types of nucleases, including zinc finger nucleases (ZFNs), transcription activator-like effector nucleases (TALENs), and the clustered, regularly interspaced, short palindromic repeat (CRISPR)-associated endonuclease Cas9 have been harnessed to introduce precise and specific genome sequence change at virtually any genome locus of interest. The therapeutic relevance of genome editing, however, is challenged by the safe and efficient delivery of nuclease into targeted cells ex vivo and in vivo. This symposium will cover the fundamentals, perspective and challenge of genome editing, and highlight the recent advances that have been made on non-viral delivery of genome-editing nucleases for therapy.

Abstracts

  • 10:30:00 AM 353. Dendrimer-based Lipid Nanoparticles Deliver Therapeutic FAH mRNA to Normalize Liver Function and Extend Survival in a Mouse Model of Hepatorenal Tyrosinemia Type I, D. Siegwart*; UT Southwestern Medical Center, Dallas, TX, USA

  • 10:45:00 AM 354. Non-viral Intracellular mRNA Delivery in Human Primary Cytotoxic T Lymphocytes Using Synthetic Lipidoid Nanoparticles, X. Zhao*, Q. Xu; Tufts University, MA, MA, USA

  • 11:00:00 AM 355. Carboxylated Branched Poly(B-amino ester) Nanoparticles Enable Robust Intracellular Protein Delivery and CRISPR/Cas9 Gene Editing, Y. Rui*, D. Wilson, K. Sanders, J. Green; Johns Hopkins University School of Medicine, Baltimore, MD, USA

  • 11:15:00 AM 356. Polymer Nanoparticles for Secreted TRAIL Cancer Therapy, H. Vaughan*, C. Zamboni, N. Radant, P. Bhardwaj, D. Francisco, J. Green; Johns Hopkins University, Baltimore, MD, USA

  • 11:30:00 AM 357. Combinatorial Therapy of siMDR1 and Doxorubicin to Overcome Drug Resistance in Breast Cancer Models, J. Lee*(1), D. Oglesby(1), W. Cornett(2); (1)Clemson University, Clemson, SC, USA, (2)Greenville Health System, Greenville, SC, USA

  • 11:45:00 AM 358. Flash Nanocomplexation as a Scalable Production Method for Therapeutic pDNA/lPEI Nanoparticles, H.-w. Liu*(1,2), Y. Hu(3), I. Minn(4), H.-Q. Mao(1,2,3,5); (1)Johns Hopkins University Whiting School of Engineering, Baltimore, MD, USA, (2)Johns Hopkins University INBT, Baltimore, MD, USA, (3)Johns Hopkins University School of Medicine, Baltimore, MD,

  • 12:00:00 PM 359. Characterization of Long-term Stability of PgP/pGFP Polyplexes with Varying Cryoprotectants, J.S. Lee*(1), J. Woo(1,2), K.-T. Kim(1), C. Macks(1); (1)Clemson University, Clemson, SC, USA, (2)Johns Hopkins University, Baltimore, MD, USA

  • 12:15:00 PM 360. Dendritic Lipopeptides for High Gene Transfection Efficiency with Structure Optimization by Molecular Dynamic Simulation, H. Liang, III*, X. Chen, A. Hu, R. Jin, K. Wang, Y. Nie; National Engineering Research Center for Biomaterials, Sichuan University, Chengdu, China