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Nucleic Acids Research|February 10, 2017
Phosphorylation-specific status of RNAi triggers in pharmacokinetic and biodistribution analysesVladimir S Trubetskoy, Jacob B Griffin, Anthony L Nicholas, et al.
The Journal of Gene Medicine|May 26, 2006
Mechanism of plasmid delivery by hydrodynamic tail vein injection. I. Hepatocyte uptake of various moleculesMagdolna G Sebestyén, Vladimir G Budker, Tatiana Budker, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|April 7, 2011
Current status of pharmaceutical and genetic therapeutic approaches to treat DMDChristophe Pichavant, Annemieke Aartsma-Rus, Paula R Clemens, et al.
Pediatrics|July 3, 2003
Prospective diagnosis of 2-methylbutyryl-CoA dehydrogenase deficiency in the Hmong population by newborn screening using tandem mass spectrometryDietrich Matern, Miao He, Susan A Berry, et al.
Human Gene Therapy|February 23, 2011
Dose response in rodents and nonhuman primates after hydrodynamic limb vein delivery of naked plasmid DNAChristine I Wooddell, Julia O Hegge, Guofeng Zhang, et al.
American Journal of Medical Genetics. Part A|October 18, 2008
The role of molecular testing and enzyme analysis in the management of hypomorphic citrullinemiaDavid P Dimmock, Pamela Trapane, Annette Feigenbaum, et al.
Molecular Cancer Therapeutics|October 29, 2017
HIF2α-Targeted RNAi Therapeutic Inhibits Clear Cell Renal Cell CarcinomaSo C Wong, Weijun Cheng, Holly Hamilton, et al.
Molecular Therapy : the Journal of the American Society of Gene Therapy|February 27, 2013
Hepatocyte-targeted RNAi therapeutics for the treatment of chronic hepatitis B virus infectionChristine I Wooddell, David B Rozema, Markus Hossbach, et al.
Nucleic Acids Research|September 2, 2006
Transcriptional and phenotypic comparisons of Ppara knockout and siRNA knockdown miceAngus T De Souza, Xudong Dai, Andrew G Spencer, et al.
The Journal of Pediatrics|March 6, 2009
Efficacy of sapropterin dihydrochloride in increasing phenylalanine tolerance in children with phenylketonuria: a phase III, randomized, double-blind, placebo-controlled studyFriedrich K Trefz, Barbara K Burton, Nicola Longo, et al.
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