J
Jason H. Williams
Researcher at Drexel University
Publications - 8
Citations - 499
Jason H. Williams is an academic researcher from Drexel University. The author has contributed to research in topics: Duchenne muscular dystrophy & Exon skipping. The author has an hindex of 7, co-authored 8 publications receiving 475 citations. Previous affiliations of Jason H. Williams include University of Melbourne.
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Journal ArticleDOI
Polymersome delivery of siRNA and antisense oligonucleotides.
Younghoon Kim,Manorama Tewari,J. David Pajerowski,Shenshen Cai,Shamik Sen,Jason H. Williams,Shashank R. Sirsi,Gordon J. Lutz,Dennis E. Discher +8 more
TL;DR: In this paper, the authors demonstrate loading and functional delivery of RNAi and AON with non-ionic, nano-transforming polymersomes, which are taken up passively by cultured cells after which the vesicles transform into micelles that allow endolysosomal escape and delivery of either siRNA into cytosol for mRNA knockdown or else AON into the nucleus for exon skipping within pre-mRNA.
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Oligonucleotide-Mediated Survival of Motor Neuron Protein Expression in CNS Improves Phenotype in a Mouse Model of Spinal Muscular Atrophy
Jason H. Williams,Rebecca C. Schray,Carlyn A. Patterson,Semira O. Ayitey,Melanie K. Tallent,Gordon J. Lutz +5 more
TL;DR: It is shown that periodic intracerebroventricular delivery of this AO resulted in increased SMN expression in brain and spinal cord to as much as 50% of the level of healthy littermates, and led to a concomitant improvement in bodyweight throughout the lifespan of the SMA animals.
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Induction of dystrophin expression by exon skipping in mdx mice following intramuscular injection of antisense oligonucleotides complexed with PEG-PEI copolymers.
TL;DR: It is concluded that low Mw PEI2000(PEG550) copolymers function as high-capacity, nontoxic AO carriers suitable for in vivo transfection of skeletal muscle and are promising compounds for potential use in molecular therapy of DMD.
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Functionalized PEG-PEI copolymers complexed to exon-skipping oligonucleotides improve dystrophin expression in mdx mice
Shashank R. Sirsi,Rebecca C. Schray,Xiangying Guan,Nicole M. Lykens,Jason H. Williams,Michelle L. Erney,Gordon J. Lutz +6 more
TL;DR: Data indicate that TAT-modified PEG-PEI copolymers are effective carriers for delivery of ESOs to skeletal muscle and are promising compounds for the therapeutic treatment of DMD.
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Poly(ethylene imine)-poly(ethylene glycol) copolymers facilitate efficient delivery of antisense oligonucleotides to nuclei of mature muscle cells of mdx mice.
TL;DR: It is concluded that PEG-PEI copolymers represent high-capacity, nontoxic carriers for efficient delivery of AO to nuclei of mature myofibers.