Advances in the delivery of RNA therapeutics: from concept to clinical reality. (Q38706445)

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28655327%20AND%20SRC:MED&resulttype=core&format=json

23 March 2020

review article

3 references

29 October 2020

15 July 2021

Advances in the delivery of RNA therapeutics: from concept to clinical reality (English)

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28655327%20AND%20SRC:MED&resulttype=core&format=json

antisense oligonucleotide

23 March 2020

main subject

0 references

0 references

0 references

1 reference

17 September 2019

biomedical investigative technique

1 reference

19 September 2019

1 reference

29 October 2020

1 reference

15 July 2021

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28655327%20AND%20SRC:MED&resulttype=core&format=json

23 March 2020

James C Kaczmarek

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28655327%20AND%20SRC:MED&resulttype=core&format=json

23 March 2020

Daniel G Anderson

series ordinal

1 reference

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23 March 2020

language of work or name

English

0 references

publication date

27 June 2017

6 references

25 June 2019

17 September 2019

19 September 2019

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28655327%20AND%20SRC:MED&resulttype=core&format=json

23 March 2020

29 October 2020

15 July 2021

6 references

25 June 2019

17 September 2019

19 September 2019

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28655327%20AND%20SRC:MED&resulttype=core&format=json

23 March 2020

29 October 2020

15 July 2021

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28655327%20AND%20SRC:MED&resulttype=core&format=json

23 March 2020

issue

1 reference

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23 March 2020

page(s)

1 reference

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Creative Commons Attribution 4.0 International

23 March 2020

5 references

25 June 2019

17 September 2019

19 September 2019

29 October 2020

Therapeutic reduction of ataxin-2 extends lifespan and reduces pathology in TDP-43 mice

15 July 2021

copyrighted

0 references

cites work

1 reference

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N1-methyl-pseudouridine in mRNA enhances translation through eIF2α-dependent and independent mechanisms by increasing ribosome density

6 September 2017

1 reference

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Administration of nucleoside-modified mRNA encoding broadly neutralizing antibody protects humanized mice from HIV-1 challenge

6 September 2017

1 reference

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Overcoming cellular barriers for RNA therapeutics

6 September 2017

1 reference

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Systemic delivery of factor IX messenger RNA for protein replacement therapy

6 September 2017

1 reference

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Therapeutic genome engineering via CRISPR-Cas systems

6 September 2017

1 reference

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Safety of antisense oligonucleotide and siRNA-based therapeutics

6 September 2017

1 reference

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Zika virus protection by a single low-dose nucleoside-modified mRNA vaccination

6 September 2017

1 reference

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Chemical and structural effects of base modifications in messenger RNA.

6 September 2017

1 reference

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Non-Viral CRISPR/Cas Gene Editing In Vitro and In Vivo Enabled by Synthetic Nanoparticle Co-Delivery of Cas9 mRNA and sgRNA.

6 September 2017

1 reference

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Multiplex gene editing by CRISPR-Cpf1 using a single crRNA array

6 September 2017

1 reference

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Cap analogs modified with 1,2-dithiodiphosphate moiety protect mRNA from decapping and enhance its translational potential

6 September 2017

1 reference

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In vivo genome editing via CRISPR/Cas9 mediated homology-independent targeted integration

6 September 2017

1 reference

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Efficacy and immunogenicity of unmodified and pseudouridine-modified mRNA delivered systemically with lipid nanoparticles in vivo.

6 September 2017

1 reference

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A multifunctional AAV-CRISPR-Cas9 and its host response

6 September 2017

1 reference

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RNA therapeutics: RNAi and antisense mechanisms and clinical applications

6 September 2017

1 reference

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Dendrimer-RNA nanoparticles generate protective immunity against lethal Ebola, H1N1 influenza, and Toxoplasma gondii challenges with a single dose

6 September 2017

1 reference

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Therapeutic efficacy in a hemophilia B model using a biosynthetic mRNA liver depot system

6 September 2017

1 reference

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Systemic RNA delivery to dendritic cells exploits antiviral defence for cancer immunotherapy

6 September 2017

1 reference

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Coming of age: ten years of next-generation sequencing technologies

6 September 2017

1 reference

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MicroRNA therapeutics: Discovering novel targets and developing specific therapy

6 September 2017

1 reference

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

6 September 2017

1 reference

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

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5485616

Effects of Chemically Modified Messenger RNA on Protein Expression.

6 September 2017

1 reference

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Bioinspired Alkenyl Amino Alcohol Ionizable Lipid Materials for Highly Potent In Vivo mRNA Delivery.

6 September 2017

1 reference

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

6 September 2017

1 reference

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A dual AAV system enables the Cas9-mediated correction of a metabolic liver disease in newborn mice

6 September 2017

1 reference

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Editing the epigenome: technologies for programmable transcription and epigenetic modulation

6 September 2017

1 reference

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RS-1 enhances CRISPR/Cas9- and TALEN-mediated knock-in efficiency

6 September 2017

1 reference

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Enhancing homology-directed genome editing by catalytically active and inactive CRISPR-Cas9 using asymmetric donor DNA.

6 September 2017

1 reference

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High-fidelity CRISPR-Cas9 nucleases with no detectable genome-wide off-target effects.

6 September 2017

1 reference

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In vivo gene editing in dystrophic mouse muscle and muscle stem cells

6 September 2017

1 reference

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In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy.

6 September 2017

1 reference

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Postnatal genome editing partially restores dystrophin expression in a mouse model of muscular dystrophy.

6 September 2017

1 reference

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Rationally engineered Cas9 nucleases with improved specificity

6 September 2017

1 reference

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Orthogonal gene knockout and activation with a catalytically active Cas9 nuclease

6 September 2017

1 reference

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Cpf1 is a single RNA-guided endonuclease of a class 2 CRISPR-Cas system

6 September 2017

1 reference

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Efficacy and safety of patisiran for familial amyloidotic polyneuropathy: a phase II multi-dose study

6 September 2017

1 reference

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CRISPRscan: designing highly efficient sgRNAs for CRISPR-Cas9 targeting in vivo

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5485616

Inhibition of nonhomologous end joining to increase the specificity of CRISPR/Cas9 genome editing

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5485616

Knocking down disease: a progress report on siRNA therapeutics

6 September 2017

1 reference

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Chemically modified guide RNAs enhance CRISPR-Cas genome editing in human primary cells

6 September 2017

1 reference

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Sequence-engineered mRNA Without Chemical Nucleoside Modifications Enables an Effective Protein Therapy in Large Animals

6 September 2017

1 reference

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RNAi therapy targeting KRAS in combination with chemotherapy for locally advanced pancreatic cancer patients

6 September 2017

1 reference

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Therapeutic genome editing: prospects and challenges

6 September 2017

1 reference

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Genome editing. The new frontier of genome engineering with CRISPR-Cas9

6 September 2017

1 reference

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

6 September 2017

1 reference

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mRNA-based therapeutics--developing a new class of drugs

6 September 2017

1 reference

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The complexity of miRNA-mediated repression

6 September 2017

1 reference

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Overcoming the challenges in administering biopharmaceuticals: formulation and delivery strategies

6 September 2017

1 reference

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Genetics and genomics of Parkinson's disease

6 September 2017

1 reference

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RNA-directed gene editing specifically eradicates latent and prevents new HIV-1 infection.

6 September 2017

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Non-viral vectors for gene-based therapy

6 September 2017

1 reference

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Permanent alteration of PCSK9 with in vivo CRISPR-Cas9 genome editing

6 September 2017

1 reference

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Engineering adeno-associated viruses for clinical gene therapy

6 September 2017

1 reference

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In vivo endothelial siRNA delivery using polymeric nanoparticles with low molecular weight

6 September 2017

1 reference

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VCAM-1 specific PEGylated SAINT-based lipoplexes deliver siRNA to activated endothelium in vivo but do not attenuate target gene expression.

6 September 2017

1 reference

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Genome editing with Cas9 in adult mice corrects a disease mutation and phenotype.

6 September 2017

1 reference

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Nucleic acid vaccines: prospects for non-viral delivery of mRNA vaccines

6 September 2017

1 reference

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Lipopeptide nanoparticles for potent and selective siRNA delivery in rodents and nonhuman primates.

6 September 2017

1 reference

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Improving CRISPR-Cas nuclease specificity using truncated guide RNAs

6 September 2017

1 reference

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Delivery materials for siRNA therapeutics

6 September 2017

1 reference

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Progress in microRNA delivery

6 September 2017

1 reference

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Modified mRNA directs the fate of heart progenitor cells and induces vascular regeneration after myocardial infarction

6 September 2017

1 reference

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A bioreducible linear poly(β-amino ester) for siRNA delivery

6 September 2017

1 reference

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Multiplex genome engineering using CRISPR/Cas systems

6 September 2017

1 reference

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Protective efficacy of in vitro synthesized, specific mRNA vaccines against influenza A virus infection

6 September 2017

1 reference

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Delivering the promise of miRNA cancer therapeutics

6 September 2017

1 reference

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Cystamine-terminated poly(beta-amino ester)s for siRNA delivery to human mesenchymal stem cells and enhancement of osteogenic differentiation

6 September 2017

1 reference

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

6 September 2017

1 reference

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The Streptococcus thermophilus CRISPR/Cas system provides immunity in Escherichia coli

6 September 2017

1 reference

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Therapeutic in vivo gene transfer for genetic disease using AAV: progress and challenges

6 September 2017

1 reference

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Exploring the Genomes of Cancer Cells: Progress and Promise

6 September 2017

1 reference

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mRNA as gene therapeutic: how to control protein expression.

6 September 2017

1 reference

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Functional genomics and rheumatoid arthritis: where have we been and where should we go?

6 September 2017

1 reference

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Endocytosis of nanomedicines.

6 September 2017

1 reference

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

6 September 2017

1 reference

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Lipid-based nanotherapeutics for siRNA delivery

6 September 2017

1 reference

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Missing pieces in understanding the intracellular trafficking of polycation/DNA complexes

6 September 2017

1 reference

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Systems medicine: the future of medical genomics and healthcare

6 September 2017

1 reference

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The RNA-induced silencing complex: a versatile gene-silencing machine

6 September 2017

1 reference

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A large-scale chemical modification screen identifies design rules to generate siRNAs with high activity, high stability and low toxicity

6 September 2017

1 reference

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Chemical modification of siRNAs for in vivo use.

6 September 2017

1 reference

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Thirty years of Alzheimer's disease genetics: the implications of systematic meta-analyses

6 September 2017

1 reference

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

6 September 2017

1 reference

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Sequence- and target-independent angiogenesis suppression by siRNA via TLR3

6 September 2017

1 reference

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Overcoming the innate immune response to small interfering RNA.

6 September 2017

1 reference

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The challenge of drugging undruggable targets in cancer: lessons learned from targeting BCL-2 family members

6 September 2017

1 reference

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Self-assembled lamellar complexes of siRNA with lipidic aminoglycoside derivatives promote efficient siRNA delivery and interference

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5485616

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

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5485616

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

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5485616

Design and development of polymers for gene delivery

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5485616

Potent and persistent in vivo anti-HBV activity of chemically modified siRNAs

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5485616

Novel "anti-reverse" cap analogs with superior translational properties

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5485616

Semi-automated synthesis and screening of a large library of degradable cationic polymers for gene delivery

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5485616

Gene silencing in mammals by small interfering RNAs

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5485616

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5485616

Fomivirsen: clinical pharmacology and potential drug interactions

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5485616

Importance of nucleotide sequence and chemical modifications of antisense oligonucleotides

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5485616

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

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5485616

A versatile vector for gene and oligonucleotide transfer into cells in culture and in vivo: polyethylenimine

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5485616

Cornerstones of CRISPR-Cas in drug discovery and therapy

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5485616

CRISPR-Based Technologies for the Manipulation of Eukaryotic Genomes

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5485616

In vitro and ex vivo strategies for intracellular delivery.

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5485616

CRISPR-Cas9 gene editing: Delivery aspects and therapeutic potential

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5485616

Methods for Optimizing CRISPR-Cas9 Genome Editing Specificity

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5485616

From the RNA world to the clinic

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5485616

Recent innovations in mRNA vaccines

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5485616

Optimization of Lipid Nanoparticle Formulations for mRNA Delivery in Vivo with Fractional Factorial and Definitive Screening Designs

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5485616

N(1)-methylpseudouridine-incorporated mRNA outperforms pseudouridine-incorporated mRNA by providing enhanced protein expression and reduced immunogenicity in mammalian cell lines and mice

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5485616

Pharmacological inhibition of DNA-PK stimulates Cas9-mediated genome editing

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5485616

Mammalian synthetic circuits with RNA binding proteins for RNA-only delivery.

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5485616

Cellular uptake and trafficking of antisense oligonucleotides

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5485616

Genome Editing for the Study of Cardiovascular Diseases

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5485616

Development of lipidoid-siRNA formulations for systemic delivery to the liver

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5485616

Lipid Nanoparticle Assisted mRNA Delivery for Potent Cancer Immunotherapy

6 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5485616

Antibody-mediated targeting of siRNA via the human insulin receptor using avidin-biotin technology

14 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5485616

Efficient in vivo delivery of siRNA to the liver by conjugation of alpha-tocopherol

14 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5485616

Evaluation of polyamidoamine dendrimer/alpha-cyclodextrin conjugate (generation 3, G3) as a novel carrier for small interfering RNA (siRNA).

14 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5485616

mRNA transfection of dendritic cells: synergistic effect of ARCA mRNA capping with Poly(A) chains in cis and in trans for a high protein expression level

14 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5485616

Positional effect of chemical modifications on short interference RNA activity in mammalian cells

14 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5485616

Antisense-oligonucleotide therapy

14 September 2017

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5485616

25 September 2017

The 'anti-hype' vaccine

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5485616

Polymer-Lipid Nanoparticles for Systemic Delivery of mRNA to the Lungs

23 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5485616

Surface charge tunability as a powerful strategy to control electrostatic interaction for high efficiency silencing, using tailored oligopeptide-modified poly(beta-amino ester)s (PBAEs).

23 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5485616

Codon optimality is a major determinant of mRNA stability

23 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5485616

Systemic delivery of therapeutic small interfering RNA using a pH-triggered amphiphilic poly-L-lysine nanocarrier to suppress prostate cancer growth in mice.

23 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5485616

RNA interference in vitro and in vivo using a novel chitosan/siRNA nanoparticle system.

23 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5485616

siRNA function in RNAi: a chemical modification analysis

23 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5485616

Introduction of double-strand breaks into the genome of mouse cells by expression of a rare-cutting endonuclease

23 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5485616

Direct gene transfer into mouse muscle in vivo

23 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5485616

Antisense oligonucleotide therapy for spinocerebellar ataxia type 2.

23 June 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5485616

Direct Cytosolic Delivery of CRISPR/Cas9-Ribonucleoprotein for Efficient Gene Editing.

31 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5485616

Systemic administration of miRNA mimics by liposomal delivery system in animal model of colorectal carcinoma.

31 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5485616

31 August 2018

Exon Skipping Therapy

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5485616

Translation efficiency of mRNAs is increased by antisense oligonucleotides targeting upstream open reading frames

31 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5485616

PGMA-Based Star-Like Polycations with Plentiful Hydroxyl Groups Act as Highly Efficient miRNA Delivery Nanovectors for Effective Applications in Heart Diseases

31 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5485616

In vivo genome editing using nuclease-encoding mRNA corrects SP-B deficiency

31 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5485616

Safety and efficacy of RNAi therapy for transthyretin amyloidosis

31 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5485616

Eteplirsen for the treatment of Duchenne muscular dystrophy

31 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5485616

Mechanism of polyplex- and lipoplex-mediated delivery of nucleic acids: real-time visualization of transient membrane destabilization without endosomal lysis

31 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5485616

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

31 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5485616

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

31 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5485616

Antisense oligonucleotide directed to human apolipoprotein B-100 reduces lipoprotein(a) levels and oxidized phospholipids on human apolipoprotein B-100 particles in lipoprotein(a) transgenic mice

31 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5485616

Modification of antigen-encoding RNA increases stability, translational efficacy, and T-cell stimulatory capacity of dendritic cells.

31 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5485616

Therapeutic silencing of an endogenous gene by systemic administration of modified siRNAs

31 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5485616

Steroid and lipid conjugates of siRNAs to enhance cellular uptake and gene silencing in liver cells

31 August 2018

1 reference

https://eutils.ncbi.nlm.nih.gov/entrez/eutils/elink.fcgi?dbfrom=pmc&linkname=pmc_refs_pubmed&retmode=json&id=5485616

Degradable Poly(β-amino esters): Synthesis, Characterization, and Self-Assembly with Plasmid DNA

31 August 2018

1 reference

Crossref

https://api.crossref.org/works/10.1186%2FS13073-017-0450-0

A phase I/IIa immunotherapy trial of HIV-1-infected patients with Tat, Rev and Nef expressing dendritic cells followed by treatment interruption

21 January 2018

1 reference

Crossref

https://api.crossref.org/works/10.1186%2FS13073-017-0450-0

Spinal muscular atrophy approval boosts antisense drugs

21 January 2018

1 reference

12 December 2020

Rapid discovery of potent siRNA-containing lipid nanoparticles enabled by controlled microfluidic formulation

1 reference

12 December 2020

Alnylam terminates revusiran program, stock plunges

1 reference

12 December 2020

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

1 reference

12 December 2020

10.1186/S13073-017-0450-0

Identifiers

DOI

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28655327%20AND%20SRC:MED&resulttype=core&format=json

Dimensions Publication ID

23 March 2020

0 references

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28655327%20AND%20SRC:MED&resulttype=core&format=json

23 March 2020

PubMed publication ID

28655327

1 reference

https://www.ebi.ac.uk/europepmc/webservices/rest/search?query=EXT_ID:28655327%20AND%20SRC:MED&resulttype=core&format=json