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Preclinical and clinical development of siRNA-based therapeutics
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PubMed
review article
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Europe PubMed Central
title
Preclinical and clinical development of siRNA-based therapeutics
(English)
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PubMed
author name string
Gulnihal Ozcan
series ordinal
1
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Crossref
Bulent Ozpolat
series ordinal
2
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stated in
Crossref
Robert L. Coleman
series ordinal
3
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Crossref
Anil K. Sood
series ordinal
4
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Crossref
Gabriel Lopez-Berestein
series ordinal
5
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Crossref
language of work or name
English
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stated in
PubMed
publication date
29 June 2015
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stated in
PubMed
published in
Advanced Drug Delivery Reviews
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PubMed
volume
87
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PubMed
page(s)
108-19
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PubMed
cites work
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Differential regulation of EphA2 in normal and malignant cells
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Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells
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Potent and specific genetic interference by double-stranded RNA in Caenorhabditis elegans
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cDNA cloning and characterization of eck, an epithelial cell receptor protein-tyrosine kinase in the eph/elk family of protein kinases
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Activation of the EphA2 tyrosine kinase stimulates the MAP/ERK kinase signaling cascade
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Mechanisms of gene silencing by double-stranded RNA
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Overexpression of protein tyrosine kinases in human esophageal cancer
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siRNAs can function as miRNAs
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Correlating animal and human phase Ia/Ib clinical data with CALAA-01, a targeted, polymer-based nanoparticle containing siRNA.
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2'-OMe-phosphorodithioate-modified siRNAs show increased loading into the RISC complex and enhanced anti-tumour activity
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Liposomal siRNA nanocarriers for cancer therapy
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Effect of an RNA interference drug on the synthesis of proprotein convertase subtilisin/kexin type 9 (PCSK9) and the concentration of serum LDL cholesterol in healthy volunteers: a randomised, single-blind, placebo-controlled, phase 1 trial
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In vitro and in vivo efficacy of SYL040012, a novel siRNA compound for treatment of glaucoma
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29 September 2017
Therapeutic Silencing of Bcl-2 by Systemically Administered siRNA Nanotherapeutics Inhibits Tumor Growth by Autophagy and Apoptosis and Enhances the Efficacy of Chemotherapy in Orthotopic Xenograft Models of ER (-) and ER (+) Breast Cancer
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Nanoparticle-Based Delivery of RNAi Therapeutics: Progress and Challenges.
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Long noncoding RNAs: cellular address codes in development and disease
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Enhancing chemotherapy response with sustained EphA2 silencing using multistage vector delivery.
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Highly specific targeting of the TMPRSS2/ERG fusion gene using liposomal nanovectors
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Phase 1 dose-escalation study of a siRNA targeting the RTP801 gene in age-related macular degeneration patients
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RNA-based therapeutics: current progress and future prospects.
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29 September 2017
Current progress of siRNA/shRNA therapeutics in clinical trials
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RNA interference in the clinic: challenges and future directions
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Designing highly active siRNAs for therapeutic applications.
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29 September 2017
Delivery of siRNA therapeutics: barriers and carriers
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Recognizing and avoiding siRNA off-target effects for target identification and therapeutic application
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First-in-human mutation-targeted siRNA phase Ib trial of an inherited skin disorder.
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Selection of hyperfunctional siRNAs with improved potency and specificity
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siRNAs: their potential as therapeutic agents--Part I. Designing of siRNAs
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All-in-one target-cell-specific magnetic nanoparticles for simultaneous molecular imaging and siRNA delivery
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29 September 2017
Selective protection of nuclease-sensitive sites in siRNA prolongs silencing effect
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29 September 2017
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29 September 2017
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29 September 2017
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29 September 2017
Effect of interleukin-8 gene silencing with liposome-encapsulated small interfering RNA on ovarian cancer cell growth
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Functional dissection of siRNA sequence by systematic DNA substitution: modified siRNA with a DNA seed arm is a powerful tool for mammalian gene silencing with significantly reduced off-target effect.
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29 September 2017
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29 September 2017
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29 September 2017
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29 September 2017
Materializing the potential of small interfering RNA via a tumor-targeting nanodelivery system.
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29 September 2017
Administration in non-human primates of escalating intravenous doses of targeted nanoparticles containing ribonucleotide reductase subunit M2 siRNA.
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29 September 2017
Quantum-dot based nanoparticles for targeted silencing of HER2/neu gene via RNA interference
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EphA2 overexpression is associated with angiogenesis in ovarian cancer
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29 September 2017
Efficacy and antivascular effects of EphA2 reduction with an agonistic antibody in ovarian cancer
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29 September 2017
Focal adhesion kinase targeting using in vivo short interfering RNA delivery in neutral liposomes for ovarian carcinoma therapy.
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29 September 2017
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29 September 2017
Off-target effects by siRNA can induce toxic phenotype
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29 September 2017
EphA2 as a target for ovarian cancer therapy
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29 September 2017
Antitumor activity of poly(ethylene glycol)-camptothecin conjugate: the inhibition of tumor growth in vivo
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29 September 2017
Therapeutic EphA2 gene targeting in vivo using neutral liposomal small interfering RNA delivery
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29 September 2017
The clinical significance of EphA2 and Ephrin A-1 in epithelial ovarian carcinomas
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29 September 2017
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29 September 2017
EphA2 expression is associated with aggressive features in ovarian carcinoma
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29 September 2017
Atelocollagen-mediated synthetic small interfering RNA delivery for effective gene silencing in vitro and in vivo
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29 September 2017
siRNA Selection Server: an automated siRNA oligonucleotide prediction server
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29 September 2017
Many commonly used siRNAs risk off-target activity
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29 September 2017
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29 September 2017
In vivo tumor growth inhibition and biodistribution studies of locked nucleic acid (LNA) antisense oligonucleotides
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29 September 2017
Protein tyrosine kinases in malignant melanoma
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29 September 2017
First line chemotherapy with carboplatin plus paclitaxel in advanced ovarian cancer--a new standard of care?
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retrieved
29 September 2017
Cyclophosphamide and cisplatin compared with paclitaxel and cisplatin in patients with stage III and stage IV ovarian cancer
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4504743
retrieved
29 September 2017
Altered gene expression in plants due to trans interactions between homologous genes
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4504743
retrieved
29 September 2017
siPools: highly complex but accurately defined siRNA pools eliminate off-target effects.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4504743
retrieved
2 June 2018
RNAi-based treatment for neovascular age-related macular degeneration by Sirna-027.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4504743
retrieved
2 June 2018
Antisense oligonucleotides containing locked nucleic acid improve potency but cause significant hepatotoxicity in animals
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4504743
retrieved
2 June 2018
Position-specific chemical modification of siRNAs reduces "off-target" transcript silencing
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4504743
retrieved
2 June 2018
Widespread siRNA "off-target" transcript silencing mediated by seed region sequence complementarity
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4504743
retrieved
2 June 2018
Distinct and overlapping expression patterns of ligands for Eph-related receptor tyrosine kinases during mouse embryogenesis
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4504743
retrieved
2 June 2018
Safety and efficacy of RNAi therapy for transthyretin amyloidosis
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4504743
retrieved
27 November 2018
Designing functional siRNA with reduced off-target effects.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4504743
retrieved
27 November 2018
Phase I clinical development of Atu027, a siRNA formulation targeting PKN3 in patients with advanced solid tumors.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4504743
retrieved
27 November 2018
RNA interference therapy in lung transplant patients infected with respiratory syncytial virus
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4504743
retrieved
27 November 2018
Metabolite identification of small interfering RNA duplex by high-resolution accurate mass spectrometry
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4504743
retrieved
27 November 2018
Design, manufacture, and assay of the efficacy of siRNAs for gene silencing.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4504743
retrieved
27 November 2018
Therapeutic targeting of neuropilin-2 on colorectal carcinoma cells implanted in the murine liver
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4504743
retrieved
27 November 2018
Liposome-incorporated Grb2 antisense oligodeoxynucleotide increases the survival of mice bearing bcr-abl-positive leukemia xenografts.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4504743
retrieved
27 November 2018
Mechanisms and optimization of in vivo delivery of lipophilic siRNAs.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4504743
retrieved
27 November 2018
Suppression of immunostimulatory siRNA-driven innate immune activation by 2'-modified RNAs.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4504743
retrieved
27 November 2018
Targeted quantum dot conjugates for siRNA delivery
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4504743
retrieved
27 November 2018
Effect of siRNA nuclease stability on the in vitro and in vivo kinetics of siRNA-mediated gene silencing
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4504743
retrieved
27 November 2018
Chemical modification of siRNAs to improve serum stability without loss of efficacy.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4504743
retrieved
27 November 2018
RNAi-mediated gene silencing in non-human primates.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4504743
retrieved
27 November 2018
Sequence characteristics of functional siRNAs
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4504743
retrieved
27 November 2018
Designing effective siRNAs with off-target control.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4504743
retrieved
27 November 2018
Therapeutic silencing of an endogenous gene by systemic administration of modified siRNAs
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4504743
retrieved
27 November 2018
Filtering of ineffective siRNAs and improved siRNA design tool.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4504743
retrieved
27 November 2018
Improved and automated prediction of effective siRNA.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4504743
retrieved
27 November 2018
Biodistribution of phosphodiester and phosphorothioate siRNA.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4504743
retrieved
27 November 2018
Predictive value of the EphA2 receptor tyrosine kinase in lung cancer recurrence and survival.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4504743
retrieved
27 November 2018
Quantification of 2'-fluoro-2'-deoxyuridine and 2'-fluoro-2'-deoxycytidine in DNA and RNA isolated from rats and woodchucks using LC/MS/MS.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4504743
retrieved
27 November 2018
Molecular regulation of tumor cell vasculogenic mimicry by tyrosine phosphorylation: role of epithelial cell kinase (Eck/EphA2).
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4504743
retrieved
27 November 2018
EphA2 overexpression causes tumorigenesis of mammary epithelial cells.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4504743
retrieved
27 November 2018
Oxygen radical-mediated pulmonary toxicity induced by some cationic liposomes
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4504743
retrieved
27 November 2018
An evaluation of the toxicities of 2'-fluorouridine and 2'-fluorocytidine-HCl in F344 rats and woodchucks (Marmota monax).
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4504743
retrieved
27 November 2018
Overexpression of the EphA2 tyrosine kinase in prostate cancer.
1 reference
stated in
PubMed Central
reference URL
https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=4504743
retrieved
27 November 2018
Liposomal delivery of oligodeoxynucleotides
1 reference
stated in
PubMed
reference URL
https://pubmed.ncbi.nlm.nih.gov/25666164
retrieved
12 December 2020
based on heuristic
inferred from PubMed ID database lookup
Safety, pharmacokinetics, and tissue distribution of liposomal P-ethoxy antisense oligonucleotides targeted to Bcl-2
1 reference
stated in
PubMed
reference URL
https://pubmed.ncbi.nlm.nih.gov/25666164
retrieved
12 December 2020
based on heuristic
inferred from PubMed ID database lookup
Potential design rules and enzymatic synthesis of siRNAs
1 reference
stated in
PubMed
reference URL
https://pubmed.ncbi.nlm.nih.gov/25666164
retrieved
12 December 2020
based on heuristic
inferred from PubMed ID database lookup
Sequence-dependent stimulation of the mammalian innate immune response by synthetic siRNA
1 reference
stated in
PubMed
reference URL
https://pubmed.ncbi.nlm.nih.gov/25666164
retrieved
12 December 2020
based on heuristic
inferred from PubMed ID database lookup
Cellular uptake and localization of liposomal-methylphosphonate oligodeoxynucleotides
1 reference
stated in
PubMed
reference URL
https://pubmed.ncbi.nlm.nih.gov/25666164
retrieved
12 December 2020
based on heuristic
inferred from PubMed ID database lookup
Identifiers
DOI
10.1016/J.ADDR.2015.01.007
1 reference
stated in
Consolidated OpenCitations Corpus – April 2017
OpenCitations bibliographic resource ID
772538
OpenCitations bibliographic resource ID
772538
1 reference
stated in
Consolidated OpenCitations Corpus – April 2017
OpenCitations bibliographic resource ID
772538
PMC publication ID
4504743
1 reference
stated in
Consolidated OpenCitations Corpus – April 2017
OpenCitations bibliographic resource ID
772538
PubMed publication ID
25666164
1 reference
stated in
Consolidated OpenCitations Corpus – April 2017
OpenCitations bibliographic resource ID
772538
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