Molecular Medicine : An Introduction.
Jens Kurreck , Cy Aaron Stein. Easy to read, yet comprehensive, this is the perfect introduction into the molecular basis of disease and the novel treatment options that have become available. The authors, Jens Kurreck and Cy Stein, have both long-standing teaching experience on the subject, one from a biologist's angle, the other with a medical background. Together, they have produced a modern textbook for courses in Molecular Medicine that incorporates modules from immunology to signaling, from virology to gene therapy, and the latest development in personalized medicine.
Genetic Disorders. Dmytro Honcharenko 1 ,. Malgorzata Honcharenko 1 ,. Full text PDF Related articles.
Abstract In vivo bioavailability and delivery of nucleic acids to the site of action is a severe limitation in oligonucleotide ON therapeutics. Related articles Based on techniques. Sinebe et al. Development of therapeutic-grade small interfering RNAs by chemical engineering. Front Genet ; Improved silencing properties using small internally segmented interfering RNAs. Efficient regulation of VEGF expression by promoter-targeted lentiviral shRNAs based on epigenetic mechanism: a novel example of epigenetherapy.
Circ Res ;— RNA duplexes with abasic substitutions are potent and allele-selective inhibitors of huntingtin and ataxin-3 expression. Allele-selective inhibition of mutant atrophin-1 expression by duplex and single-stranded RNAs. Ligands of the asialoglycoprotein receptor for targeted gene delivery, part 1: Synthesis of and binding studies with biotinylated cluster glycosides containing N-acetylgalactosamine.
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Adv Drug Deliv Rev ;— Delivery of siRNA to the brain using a combination of nose-to-brain delivery and cell-penetrating peptide-modified nano-micelles. Biomaterials ;— Smart functional nucleic acid chimeras: enabling tissue specific RNA targeting therapy. RNA Biol ;— Complexity in the therapeutic delivery of RNAi medicines: an analytical challenge. Expert Opin Drug Deliv ;— Going beyond the liver: progress and challenges of targeted delivery of siRNA therapeutics. J Control Release ;— Fomiversen sodium approved to treat CMV retinitis. J Am Pharm Assoc Wash ;— Mipomersen, an apolipoprotein B synthesis inhibitor, for lowering of LDL cholesterol concentrations in patients with homozygous familial hypercholesterolaemia: a randomised, double-blind, placebo-controlled trial.
Lancet ;— Reciprocal effects of micro-RNA on expression of heme oxygenase-1 and hepatitis C virus genes in human hepatocytes. Gastroenterology ;— Int J Oncol ;— Potential of anti-cancer therapy based on anti-miR oligonucleotides in glioma and brain tumours. Chem Biol Drug Des ;— Alzheimers Res Ther ; Curr Mol Med ;— Nat Rev Drug Discov ;— Molecular basis of muscular dystrophies.
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Curr Opin Mol Ther ;— Splice-correcting oligonucleotides restore BTK function in X-linked agammaglobulinemia model. J Clin Invest ;— Splice-correction strategies for treatment of x-linked agammaglobulinemia. Curr Allergy Asthma Rep ; The molecular basis for Duchenne versus Becker muscular dystrophy: correlation of severity with type of deletion. Am J Hum Genet ;— Clinical trials using antisense oligonucleotides in Duchenne muscular dystrophy. Antisense oligonucleotide therapeutics for iron-sulphur cluster deficiency myopathy.
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Purine twisted-intercalating nucleic acids: a new class of anti-gene molecules resistant to potassium-induced aggregation. Triplex-forming oligonucleotides targeting c-MYC potentiate the anti-tumor activity of gemcitabine in a mouse model of human cancer.
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Cellular uptake and intracellular trafficking of oligonucleotides. Google Scholar. RNA G-quadruplex as supramolecular carrier for cancer-selective delivery. The study of genes and signal transduction pathways involved in mustard lung injury: A gene therapy approach. Sara C. Humphreys , Mai B. Thayer , Iain D. Campuzano , Chawita Netirojjanakul , and Brooke M. Arylation Chemistry for Bioconjugation. The ability of locked nucleic acid oligonucleotides to pre-structure the double helix: A molecular simulation and binding study.
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Non-coding RNA in C9orfrelated amyotrophic lateral sclerosis and frontotemporal dementia: A perfect storm of dysfunction.