Overcoming cellular barriers for RNA therapeutics (Q30235525)

11 June 2017

Overcoming cellular barriers for RNA therapeutics (English)

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11 June 2017

Steven F Dowdy

1

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11 June 2017

27 February 2017

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11 June 2017

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11 June 2017

35

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11 June 2017

222-229

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11 June 2017

3

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Results from a phase 1 study of nusinersen (ISIS-SMN(Rx)) in children with spinal muscular atrophy

11 June 2017

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7 January 2021

Intrathecal Injections in Children With Spinal Muscular Atrophy: Nusinersen Clinical Trial Experience

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7 January 2021

A Highly Durable RNAi Therapeutic Inhibitor of PCSK9.

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7 January 2021

Primeval cells: Possible energy-generating and cell-division mechanisms

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7 January 2021

The "strong" RNA world hypothesis: fifty years old.

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7 January 2021

Progress toward synthetic cells

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7 January 2021

Cryo-electron microscopy for structure analyses of membrane proteins in the lipid bilayer.

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7 January 2021

The response of mammalian cells to double-stranded RNA

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7 January 2021

Optimized siRNA-PEG conjugates for extended blood circulation and reduced urine excretion in mice

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7 January 2021

Cellular uptake and intracellular trafficking of oligonucleotides: implications for oligonucleotide pharmacology

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7 January 2021

Experimental and computational approaches to estimate solubility and permeability in drug discovery and development settings

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7 January 2021

Mechanisms of endocytosis

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7 January 2021

The delivery of therapeutic oligonucleotides

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7 January 2021

Inhibition of Rous sarcoma viral RNA translation by a specific oligodeoxyribonucleotide

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7 January 2021

Inhibition of Rous sarcoma virus replication and cell transformation by a specific oligodeoxynucleotide

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7 January 2021

Phosphorothioate analogs of oligodeoxynucleotides: inhibitors of replication and cytopathic effects of human immunodeficiency virus

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7 January 2021

Oligodeoxynucleoside phosphoramidates and phosphorothioates as inhibitors of human immunodeficiency virus

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7 January 2021

Phosphorothioates, essential components of therapeutic oligonucleotides

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7 January 2021

Phosphorothioate oligonucleotides bind in a non sequence-specific manner to the nucleolar protein C23/nucleolin

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7 January 2021

Identification and characterization of intracellular proteins that bind oligonucleotides with phosphorothioate linkages

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7 January 2021

Solid-phase synthesis of stereoregular oligodeoxyribonucleoside phosphorothioates using bicyclic oxazaphospholidine derivatives as monomer units.

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7 January 2021

Synthesis, biophysical properties and biological activity of second generation antisense oligonucleotides containing chiral phosphorothioate linkages.

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7 January 2021

Influence of diverse chemical modifications on the ADME characteristics and toxicology of antisense oligonucleotides.

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7 January 2021

Efficient gene silencing by delivery of locked nucleic acid antisense oligonucleotides, unassisted by transfection reagents

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7 January 2021

A cytoplasmic pathway for gapmer antisense oligonucleotide-mediated gene silencing in mammalian cells

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7 January 2021

Hsp90 protein interacts with phosphorothioate oligonucleotides containing hydrophobic 2'-modifications and enhances antisense activity

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7 January 2021

Gene-targeting pharmaceuticals for single-gene disorders

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7 January 2021

Targeted delivery of antisense oligonucleotides to hepatocytes using triantennary N-acetyl galactosamine improves potency 10-fold in mice.

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7 January 2021

Disposition and Pharmacology of a GalNAc3-conjugated ASO Targeting Human Lipoprotein (a) in Mice

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7 January 2021

Design and synthesis of novel amphiphilic dendritic galactosides for selective targeting of liposomes to the hepatic asialoglycoprotein receptor.

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7 January 2021

Antisense oligonucleotide therapy for neurodegenerative disease.

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7 January 2021

Sustained therapeutic reversal of Huntington's disease by transient repression of huntingtin synthesis.

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7 January 2021

Single-stranded RNAs use RNAi to potently and allele-selectively inhibit mutant huntingtin expression.

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7 January 2021

Long pre-mRNA depletion and RNA missplicing contribute to neuronal vulnerability from loss of TDP-43.

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7 January 2021

Targeted degradation of sense and antisense C9orf72 RNA foci as therapy for ALS and frontotemporal degeneration

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7 January 2021

Gain of Toxicity from ALS/FTD-Linked Repeat Expansions in C9ORF72 Is Alleviated by Antisense Oligonucleotides Targeting GGGGCC-Containing RNAs

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7 January 2021

Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model

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7 January 2021

Antisense oligonucleotides delivered to the mouse CNS ameliorate symptoms of severe spinal muscular atrophy

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7 January 2021

Morpholino antisense oligomers: design, preparation, and properties

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7 January 2021

A chemical view of oligonucleotides for exon skipping and related drug applications

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7 January 2021

Morpholinos and their peptide conjugates: therapeutic promise and challenge for Duchenne muscular dystrophy

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7 January 2021

Systemic delivery of morpholino oligonucleotide restores dystrophin expression bodywide and improves dystrophic pathology

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7 January 2021

Dystrophin expression in the mdx mouse after localised and systemic administration of a morpholino antisense oligonucleotide

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7 January 2021

Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells

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7 January 2021

Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans

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7 January 2021

Intracellular localization and routing of miRNA and RNAi pathway components

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7 January 2021

Molecular basis of double-stranded RNA-protein interactions: structure of a dsRNA-binding domain complexed with dsRNA.

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7 January 2021

Nucleation, propagation and cleavage of target RNAs in Ago silencing complexes

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7 January 2021

From guide to target: molecular insights into eukaryotic RNA-interference machinery.

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7 January 2021

Progress toward in vivo use of siRNAs-II

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7 January 2021

siRNA and innate immunity

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7 January 2021

An RNAi therapeutic targeting antithrombin to rebalance the coagulation system and promote hemostasis in hemophilia.

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7 January 2021

Asymmetric RNA duplexes mediate RNA interference in mammalian cells.

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7 January 2021

Asymmetric shorter-duplex siRNA structures trigger efficient gene silencing with reduced nonspecific effects

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7 January 2021

Novel hydrophobically modified asymmetric RNAi compounds (sd-rxRNA) demonstrate robust efficacy in the eye.

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7 January 2021

Efficient delivery of RNAi prodrugs containing reversible charge-neutralizing phosphotriester backbone modifications.

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7 January 2021

Synthesis and Conjugation of Small Interfering Ribonucleic Neutral SiRNNs

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7 January 2021

RNAi-mediated gene silencing in non-human primates.

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7 January 2021

A combinatorial library of lipid-like materials for delivery of RNAi therapeutics

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7 January 2021

Rational design of cationic lipids for siRNA delivery

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7 January 2021

Lipid-like materials for low-dose, in vivo gene silencing

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7 January 2021

RNA interference in the clinic: challenges and future directions

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7 January 2021

Delivery materials for siRNA therapeutics

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7 January 2021

A Retrospective 30 Years After Discovery of the Enhanced Permeability and Retention Effect of Solid Tumors: Next-Generation Chemotherapeutics and Photodynamic Therapy--Problems, Solutions, and Prospects

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7 January 2021

Multivalent N-acetylgalactosamine-conjugated siRNA localizes in hepatocytes and elicits robust RNAi-mediated gene silencing

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7 January 2021

Hepatocyte-specific delivery of siRNAs conjugated to novel non-nucleosidic trivalent N-acetylgalactosamine elicits robust gene silencing in vivo

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7 January 2021

Antibody mediated in vivo delivery of small interfering RNAs via cell-surface receptors

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7 January 2021

Systematic evaluation of antibody-mediated siRNA delivery using an industrial platform of THIOMAB-siRNA conjugates.

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7 January 2021

Direct gene transfer into mouse muscle in vivo

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7 January 2021

Incorporation of pseudouridine into mRNA yields superior nonimmunogenic vector with increased translational capacity and biological stability

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7 January 2021

Expression of therapeutic proteins after delivery of chemically modified mRNA in mice

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7 January 2021

Efficient generation of human iPSCs by a synthetic self-replicative RNA.

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7 January 2021

RNA-based vaccination: sending a strong message

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7 January 2021

Enhanced Delivery and Potency of Self-Amplifying mRNA Vaccines by Electroporation in Situ

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7 January 2021

A cationic nanoemulsion for the delivery of next-generation RNA vaccines.

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7 January 2021

Self-amplifying mRNA vaccines

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7 January 2021

A Programmable Dual-RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity

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7 January 2021

Multiplex genome engineering using CRISPR/Cas systems

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7 January 2021

RNA-guided human genome engineering via Cas9

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7 January 2021

DNA interrogation by the CRISPR RNA-guided endonuclease Cas9.

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7 January 2021

In vivo genome editing using Staphylococcus aureus Cas9

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7 January 2021

Improving CRISPR-Cas nuclease specificity using truncated guide RNAs

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7 January 2021

Don’t edit the human germ line

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7 January 2021

Biotechnology. A prudent path forward for genomic engineering and germline gene modification

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7 January 2021

Efficient delivery of nuclease proteins for genome editing in human stem cells and primary cells

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7 January 2021

Therapeutic genome editing by combined viral and non-viral delivery of CRISPR system components in vivo

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7 January 2021

Efficient delivery of genome-editing proteins using bioreducible lipid nanoparticles.

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7 January 2021

Cationic lipid-mediated delivery of proteins enables efficient protein-based genome editing in vitro and in vivo

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7 January 2021

Cationic PTD/CPP-mediated macromolecular delivery: charging into the cell

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7 January 2021

The asialoglycoprotein receptor: a model for endocytic transport receptors

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7 January 2021

Visualizing lipid-formulated siRNA release from endosomes and target gene knockdown

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7 January 2021

Weak bases and ionophores rapidly and reversibly raise the pH of endocytic vesicles in cultured mouse fibroblasts

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7 January 2021

High-throughput screening identifies small molecules that enhance the pharmacological effects of oligonucleotides

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7 January 2021

Guanabenz (Wytensin™) selectively enhances uptake and efficacy of hydrophobically modified siRNAs

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7 January 2021

Hepatocyte-targeted RNAi therapeutics for the treatment of chronic hepatitis B virus infection

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7 January 2021

A role for peptides in overcoming endosomal entrapment in siRNA delivery - A focus on melittin

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7 January 2021

Enhancing Endosomal Escape for Intracellular Delivery of Macromolecular Biologic Therapeutics.

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7 January 2021

Discovery and characterization of a peptide that enhances endosomal escape of delivered proteins in vitro and in vivo.

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7 January 2021

Identifiers

DOI

10.1038/NBT.3802

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11 June 2017

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PubMed ID

28244992