Immunological and hematological toxicities challenging clinical translation of nucleic acid-based therapeutics (Q38504825)

3 September 2017

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title

Immunological and hematological toxicities challenging clinical translation of nucleic acid-based therapeutics (English)

1 reference

3 September 2017

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Marina A Dobrovolskaia

series ordinal

1

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PubMed ID

26017628

3 September 2017

Scott E McNeil

series ordinal

2

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PubMed ID

26017628

3 September 2017

language of work or name

English

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publication date

28 May 2015

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Expert Opinion on Biological Therapy

3 September 2017

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3 September 2017

15

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3 September 2017

7

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3 September 2017

1023-1048

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Nucleic acids as therapeutic agents

3 September 2017

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Liver as a target for oligonucleotide therapeutics

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Use of oligonucleotide aptamer ligands to modulate the function of immune receptors

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

A review of the issues in the pharmacokinetics and toxicology of phosphorothioate antisense oligonucleotides

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Drug-protein adducts: an industry perspective on minimizing the potential for drug bioactivation in drug discovery and development

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Aptamers as therapeutics.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Coadministration of cidofovir and smallpox vaccine reduced vaccination side effects but interfered with vaccine-elicited immune responses and immunity to monkeypox

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Stereo-enriched phosphorothioate oligodeoxynucleotides: synthesis, biophysical and biological properties

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

A phase I trial of aprinocarsen (ISIS 3521/LY900003), an antisense inhibitor of protein kinase C-alpha administered as a 24-hour weekly infusion schedule in patients with advanced cancer

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

A Phase II trial of aprinocarsen, an antisense oligonucleotide inhibitor of protein kinase C alpha, administered as a 21-day infusion to patients with advanced ovarian carcinoma.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

A phase I pharmacokinetic and pharmacodynamic study of OGX-011, a 2'-methoxyethyl antisense oligonucleotide to clusterin, in patients with localized prostate cancer

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Modulation of CpG oligodeoxynucleotide-mediated immune stimulation by locked nucleic acid (LNA).

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

CpG RNA: identification of novel single-stranded RNA that stimulates human CD14+CD11c+ monocytes

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Suppression of RNA recognition by Toll-like receptors: the impact of nucleoside modification and the evolutionary origin of RNA

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

RNA:DNA hybrids are a novel molecular pattern sensed by TLR9.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

TLR9 as a key receptor for the recognition of DNA.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Toxicology of antisense therapeutics

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Evaluation of the toxicity of ISIS 2302, a phosphorothioate oligonucleotide, in a four-week study in cynomolgus monkeys.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Inhibition of coagulation by a phosphorothioate oligonucleotide.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Comparison of the toxicity profiles of ISIS 1082 and ISIS 2105, phosphorothioate oligonucleotides, following subacute intradermal administration in Sprague-Dawley rats

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Evaluation of the toxicity of ISIS 2302, a phosphorothioate oligonucleotide, in a 4-week study in CD-1 mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Correlation of toxicity and pharmacokinetic properties of a phosphorothioate oligonucleotide designed to inhibit ICAM-1.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Preclinical evaluation of the toxicological effects of a novel constrained ethyl modified antisense compound targeting signal transducer and activator of transcription 3 in mice and cynomolgus monkeys

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

A placebo-controlled trial of ICAM-1 antisense oligonucleotide in the treatment of Crohn's disease.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Synthetic oligonucleotides: the development of antisense therapeutics

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Mixed backbone antisense oligonucleotides: design, biochemical and biological properties of oligonucleotides containing 2'-5'-ribo- and 3'-5'-deoxyribonucleotide segments

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Antisense inhibition of gene expression in cells by oligonucleotides incorporating locked nucleic acids: effect of mRNA target sequence and chimera design

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Non-CpG-containing antisense 2'-methoxyethyl oligonucleotides activate a proinflammatory response independent of Toll-like receptor 9 or myeloid differentiation factor 88.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Unique O-methoxyethyl ribose-DNA chimeric oligonucleotide induces an atypical melanoma differentiation-associated gene 5-dependent induction of type I interferon response

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

'Immunomers'--novel 3'-3'-linked CpG oligodeoxyribonucleotides as potent immunomodulatory agents

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Design, synthesis, and immunostimulatory properties of CpG DNAs containing alkyl-linker substitutions: role of nucleosides in the flanking sequences

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Immunological response to peptide nucleic acid and its peptide conjugate targeted to transactivation response (TAR) region of HIV-1 RNA genome

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Potent CpG oligonucleotides containing phosphodiester linkages: in vitro and in vivo immunostimulatory properties.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Synthetic immunostimulatory oligonucleotides in experimental and clinical practice

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Innate immune sensing and signaling of cytosolic nucleic acids

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Nucleic acids and endosomal pattern recognition: how to tell friend from foe?

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

CpG DNA as a vaccine adjuvant.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Immunotherapeutic applications of CpG oligodeoxynucleotide TLR9 agonists

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Delineation of a CpG phosphorothioate oligodeoxynucleotide for activating primate immune responses in vitro and in vivo

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Identification of CpG oligonucleotide sequences with high induction of IFN-alpha/beta in plasmacytoid dendritic cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Mechanism and function of a newly identified CpG DNA motif in human primary B cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Identification of a novel CpG DNA class and motif that optimally stimulate B cell and plasmacytoid dendritic cell functions

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Distinct CpG oligonucleotide sequences activate human gamma delta T cells via interferon-alpha/-beta

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

CpG motifs in bacterial DNA trigger direct B-cell activation

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Structural requirements for uptake and recognition of CpG oligonucleotides

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Highly immunostimulatory CpG-free oligodeoxynucleotides for activation of human leukocytes.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Divergent synthetic nucleotide motif recognition pattern: design and development of potent immunomodulatory oligodeoxyribonucleotide agents with distinct cytokine induction profiles

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Novel immunomodulatory oligonucleotides prevent development of allergic airway inflammation and airway hyperresponsiveness in asthma

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Immunomodulatory oligonucleotides containing a cytosine-phosphate-2'-deoxy-7-deazaguanosine motif as potent toll-like receptor 9 agonists

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

A dinucleotide motif in oligonucleotides shows potent immunomodulatory activity and overrides species-specific recognition observed with CpG motif

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

TLR ignores methylated RNA?

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

A phase 1 study of the BCL2-targeted deoxyribonucleic acid inhibitor (DNAi) PNT2258 in patients with advanced solid tumors

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Ribozyme- and deoxyribozyme-strategies for medical applications

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Therapeutic applications of ribozymes and riboswitches

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

An open-label, phase 2 trial of RPI.4610 (Angiozyme) in the treatment of metastatic breast cancer.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Safety and pharmacokinetic study of RPI.4610 (ANGIOZYME), an anti-VEGFR-1 ribozyme, in combination with carboplatin and paclitaxel in patients with advanced solid tumors

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

HER-2/neu is rate-limiting for ovarian cancer growth. Conditional depletion of HER-2/neu by ribozyme targeting.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Past, present, and future hepatitis C treatments

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Phase 2 gene therapy trial of an anti-HIV ribozyme in autologous CD34+ cells

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Design of antisense and triplex-forming oligonucleotides

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Delivery materials for siRNA therapeutics

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

RNAi therapies: drugging the undruggable

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Interactions between double-stranded RNA regulators and the protein kinase DAI

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Synthetic shRNAs as potent RNAi triggers.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Induction of the interferon response by siRNA is cell type- and duplex length-dependent

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Small interfering RNAs mediate sequence-independent gene suppression and induce immune activation by signaling through toll-like receptor 3.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

The first targeted delivery of siRNA in humans via a self-assembling, cyclodextrin polymer-based nanoparticle: from concept to clinic.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Tumor-targeted delivery of siRNA by self-assembled nanoparticles

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Efficient oncogene silencing and metastasis inhibition via systemic delivery of siRNA.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

An efficient and low immunostimulatory nanoparticle formulation for systemic siRNA delivery to the tumor

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Biodegradable calcium phosphate nanoparticle with lipid coating for systemic siRNA delivery

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

The co-delivery of oxaliplatin abrogates the immunogenic response to PEGylated siRNA-lipoplex.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Mechanistically probing lipid-siRNA nanoparticle-associated toxicities identifies Jak inhibitors effective in mitigating multifaceted toxic responses

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Evaluation of locked nucleic acid-modified small interfering RNA in vitro and in vivo.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

In vivo sustained release of siRNA from solid lipid nanoparticles

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Novel role for the delta-opioid receptor in hypoxic preconditioning in rat retinas

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

First-in-human mutation-targeted siRNA phase Ib trial of an inherited skin disorder.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Effective treatment of respiratory alphaherpesvirus infection using RNA interference.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Eukaryotic translation initiation factor 5A small interference RNA-liposome complexes reduce inflammation and increase survival in murine models of severe sepsis and acute lung injury

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Protection against lethal influenza virus challenge by RNA interference in vivo.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Lung delivery studies using siRNA conjugated to TAT(48-60) and penetratin reveal peptide induced reduction in gene expression and induction of innate immunity.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Cell-penetrating-peptide-mediated siRNA lung delivery

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

BDNF is necessary and sufficient for spinal respiratory plasticity following intermittent hypoxia

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Central delivery of Dicer-substrate siRNA: a direct application for pain research

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Intrathecal Raf-1-selective siRNA attenuates sustained morphine-mediated thermal hyperalgesia

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Therapeutic silencing of mutant huntingtin with siRNA attenuates striatal and cortical neuropathology and behavioral deficits

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

JAK2 and STAT3 activation contributes to neuronal damage following transient focal cerebral ischemia.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Targeting ECM-integrin interaction with liposome-encapsulated small interfering RNAs inhibits the growth of human prostate cancer in a bone xenograft imaging model

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Claudin-3 gene silencing with siRNA suppresses ovarian tumor growth and metastasis

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Folate-linked lipid-based nanoparticles for synthetic siRNA delivery in KB tumor xenografts.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

An siRNA-based microbicide protects mice from lethal herpes simplex virus 2 infection

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Durable protection from Herpes Simplex Virus-2 transmission following intravaginal application of siRNAs targeting both a viral and host gene

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Engineering mucosal RNA interference in vivo

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Targeting V600EB-Raf and Akt3 using nanoliposomal-small interfering RNA inhibits cutaneous melanocytic lesion development.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Topical matrix-based siRNA silences local gene expression in a murine wound model.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Evaluation of efficacy, biodistribution, and inflammation for a potent siRNA nanoparticle: effect of dexamethasone co-treatment

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Aptamers: multifunctional molecules for biomedical research

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Chemically modified DNA aptamers bind interleukin-6 with high affinity and inhibit signaling by blocking its interaction with interleukin-6 receptor

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Toxicity testing of the VEGF inhibitors bevacizumab, ranibizumab and pegaptanib in rats both with and without prior retinal ganglion cell damage.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

A phase II trial of AS1411 (a novel nucleolin-targeted DNA aptamer) in metastatic renal cell carcinoma

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Inhibition of tissue factor pathway inhibitor by the aptamer BAX499 improves clotting of hemophilic blood and plasma.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

AS1411 aptamer tagged PLGA-lecithin-PEG nanoparticles for tumor cell targeting and drug delivery.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Clinical optimization of antigen specific modulation of type 1 diabetes with the plasmid DNA platform

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Efficacy trial of a DNA/rAd5 HIV-1 preventive vaccine

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Absence of systemic toxicity changes following intramuscular administration of novel pSG2.HIVconsv DNA, ChAdV63.HIVconsv and MVA.HIVconsv vaccines to BALB/c mice

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Safety, tolerability, and immunogenicity of repeated doses of dermavir, a candidate therapeutic HIV vaccine, in HIV-infected patients receiving combination antiretroviral therapy: results of the ACTG 5176 trial

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Phase I trial of a formulated IL-12 plasmid in combination with carboplatin and docetaxel chemotherapy in the treatment of platinum-sensitive recurrent ovarian cancer

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Spermine-alt-poly(ethylene glycol) polyspermine as a safe and efficient aerosol gene carrier for lung cancer therapy

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Marker-free plasmids for biotechnological applications - implications and perspectives.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Generation of human induced pluripotent stem cells by simple transient transfection of plasmid DNA encoding reprogramming factors

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Induction of pluripotent stem cells from mouse embryonic and adult fibroblast cultures by defined factors

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Pharmacological Characterization of a Novel ENaCα siRNA (GSK2225745) With Potential for the Treatment of Cystic Fibrosis

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

In vivo comparison of local versus systemic delivery of immunostimulating siRNA in HPV-driven tumours.

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Drug delivery-mediated control of RNA immunostimulation

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Toll-like receptor (TLR) 3 immune modulation by unformulated small interfering RNA or DNA and the role of CD14 (in TLR-mediated effects).

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

Canonical and non-canonical barriers facing antimiR cancer therapeutics

21 January 2018

1 reference

https://api.crossref.org/works/10.1517%2F14712598.2015.1014794

10.1517/14712598.2015.1014794

21 January 2018

Identifiers

DOI

1 reference

3 September 2017

1 reference

PubMed ID

26017628