标题
Current Advances in RNA Therapeutics for Human Diseases
作者
关键词
-
出版物
INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
Volume 23, Issue 5, Pages 2736
出版商
MDPI AG
发表日期
2022-03-01
DOI
10.3390/ijms23052736
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注意：仅列出部分参考文献，下载原文获取全部文献信息。- HIPK3 Inhibition by Exosomal hsa-miR-101-3p Is Related to Metabolic Reprogramming in Colorectal Cancer
- (2022) Lihuiping Tao et al. Frontiers in Oncology
- Lumasiran: First Approval
- (2021) Lesley J. Scott et al. DRUGS
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- (2021) Rajan Singh et al. GASTROENTEROLOGY
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- (2021) Cassandra Aldrich et al. VACCINE
- The administration of antisense oligonucleotide golodirsen reduces pathological regeneration in patients with Duchenne muscular dystrophy
- (2021) Dominic Scaglioni et al. Acta Neuropathologica Communications
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- (2021) Yvette N. Lamb DRUGS
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- (2021) Liang Chang et al. Translational Cancer Research
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- (2021) Matt Shirley DRUGS
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- (2021) Yahiya Y. Syed DRUGS
- In vivo CRISPR base editing of PCSK9 durably lowers cholesterol in primates
- (2021) Kiran Musunuru et al. NATURE
- Spatial mapping and immunomodulatory role of the OX40/OX40L pathway in human non-small cell lung cancer.
- (2021) Angelo Porciuncula et al. CLINICAL CANCER RESEARCH
- Successful defibrotide treatment of a patient with veno-occlusive disease after living-donor liver transplantation
- (2021) Tae Beom Lee et al. MEDICINE
- Safety, tolerability, and pharmacokinetics of casimersen in patients with D uchenne muscular dystrophy amenable to exon 45 skipping: A randomized, double‐blind, placebo‐controlled, dose‐titration trial
- (2021) Kathryn R. Wagner et al. MUSCLE & NERVE
- Noncoding RNA therapeutics — challenges and potential solutions
- (2021) Melanie Winkle et al. NATURE REVIEWS DRUG DISCOVERY
- Pre-existing immunity to cytomegalovirus in macaques influences human CMV vaccine responses in preclinical models
- (2021) Helen Webster et al. VACCINE
- Non-Hodgkin lymphomas and ionizing radiation: case report and review of the literature
- (2021) Inge Schmitz-Feuerhake et al. ANNALS OF HEMATOLOGY
- Distinct roles of miR-34 family members on suppression of lung squamous cell carcinoma
- (2021) Dangze Sun et al. BIOMEDICINE & PHARMACOTHERAPY
- On the cusp of cures: Breakthroughs in Batten disease research
- (2021) Jon J. Brudvig et al. CURRENT OPINION IN NEUROBIOLOGY
- MicroRNA 122 Affects both the Initiation and the Maintenance of Hepatitis C Virus Infections
- (2021) Mamata Panigrahi et al. JOURNAL OF VIROLOGY
- Life of RISC: Formation, action, and degradation of RNA-induced silencing complex
- (2021) Hiro-oki Iwakawa et al. MOLECULAR CELL
- Innovative Therapeutic Approaches for Huntington’s Disease: From Nucleic Acids to GPCR-Targeting Small Molecules
- (2021) Hidetoshi Komatsu Frontiers in Cellular Neuroscience
- New Frontiers in the Treatment of Homozygous Familial Hypercholesterolemia
- (2021) Arturo Cesaro et al. Heart Failure Clinics
- Combined inhibition of CXCL12 and PD-1 in MSS colorectal and pancreatic cancer: modulation of the microenvironment and clinical effects
- (2021) Meggy Suarez-Carmona et al. Journal for ImmunoTherapy of Cancer
- Genetic Modifying Factors of Cystic Fibrosis Phenotype: A Challenge for Modern Medicine
- (2021) Lăcrămioara Ionela Butnariu et al. Journal of Clinical Medicine
- mRNA Vaccine Protects against Zika Virus
- (2021) Lex G. Medina-Magües et al. Vaccines
- Role of microRNAs in Disorders of Gut–Brain Interactions: Clinical Insights and Therapeutic Alternatives
- (2021) Rajan Singh et al. Journal of Personalized Medicine
- Efficacy and safety of the CVnCoV SARS-CoV-2 mRNA vaccine candidate in ten countries in Europe and Latin America (HERALD): a randomised, observer-blinded, placebo-controlled, phase 2b/3 trial
- (2021) Peter G Kremsner et al. LANCET INFECTIOUS DISEASES
- Keloid and Hypertrophic Scar Formation, Prevention, and Management
- (2020) Gregory Grabowski et al. JOURNAL OF THE AMERICAN ACADEMY OF ORTHOPAEDIC SURGEONS
- Role of oncogenic KRAS in the diagnosis, prognosis and treatment of pancreatic cancer
- (2020) Louis Buscail et al. Nature Reviews Gastroenterology & Hepatology
- Modeling clear cell renal cell carcinoma and therapeutic implications
- (2020) Melissa M. Wolf et al. ONCOGENE
- Preclinical development of a miR-132 inhibitor for heart failure treatment
- (2020) Ariana Foinquinos et al. Nature Communications
- RNA-Based Therapeutics: From Antisense Oligonucleotides to miRNAs
- (2020) Sarah Bajan et al. Cells
- Safety, Tolerability, and Efficacy of Viltolarsen in Boys With Duchenne Muscular Dystrophy Amenable to Exon 53 Skipping
- (2020) Paula R. Clemens et al. JAMA Neurology
- Alport Syndrome: Achieving Early Diagnosis and Treatment
- (2020) Clifford E. Kashtan AMERICAN JOURNAL OF KIDNEY DISEASES
- Single‐Dose Pharmacokinetics and Pharmacodynamics of Transthyretin Targeting N‐acetylgalactosamine–Small Interfering Ribonucleic Acid Conjugate, Vutrisiran, in Healthy Subjects
- (2020) Bahru A. Habtemariam et al. CLINICAL PHARMACOLOGY & THERAPEUTICS
- An RNA vaccine drives immunity in checkpoint-inhibitor-treated melanoma
- (2020) Ugur Sahin et al. NATURE
- Advances in oligonucleotide drug delivery
- (2020) Thomas C. Roberts et al. NATURE REVIEWS DRUG DISCOVERY
- Phase 3 Trial of RNAi Therapeutic Givosiran for Acute Intermittent Porphyria
- (2020) Manisha Balwani et al. NEW ENGLAND JOURNAL OF MEDICINE
- Antisense oligonucleotide modulation of non-productive alternative splicing upregulates gene expression
- (2020) Kian Huat Lim et al. Nature Communications
- Efficiency and Target Derepression of Anti-miR-92a: Results of a First in Human Study
- (2020) Wesley Tyler Abplanalp et al. Nucleic Acid Therapeutics
- Intratumoral interleukin-12 mRNA therapy promotes TH1 transformation of the tumor microenvironment
- (2020) Susannah L Hewitt et al. CLINICAL CANCER RESEARCH
- CDR132L improves systolic and diastolic function in a large animal model of chronic heart failure
- (2020) Sandor Batkai et al. EUROPEAN HEART JOURNAL
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- (2020) Alejandro Da Silva Sanchez et al. HUMAN GENE THERAPY
- Synthetic mRNA Encoding VEGF-A in Patients Undergoing Coronary Artery Bypass Grafting: Design of a Phase 2a Clinical Trial
- (2020) Vesa Anttila et al. Molecular Therapy-Methods & Clinical Development
- Safety and clinical activity of intratumoral MEDI9197 alone and in combination with durvalumab and/or palliative radiation therapy in patients with advanced solid tumors
- (2020) Lillian Siu et al. Journal for ImmunoTherapy of Cancer
- Safety, Efficacy and Pharcacokinetics of Targeted Therapy with The Liposomal RNA Interference Therapeutic Atu027 Combined with Gemcitabine in Patients with Pancreatic Adenocarcinoma. A Randomized Phase Ib/IIa Study
- (2020) Beate Schultheis et al. Cancers
- Cobomarsen, an Oligonucleotide Inhibitor of miR-155, Slows DLBCL Tumor Cell Growth In Vitro and In Vivo
- (2020) Eleni Anastasiadou et al. CLINICAL CANCER RESEARCH
- Efficacy and Safety of the mRNA-1273 SARS-CoV-2 Vaccine
- (2020) Lindsey R. Baden et al. NEW ENGLAND JOURNAL OF MEDICINE
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- (2020) Fernando P. Polack et al. NEW ENGLAND JOURNAL OF MEDICINE
- Current and emerging therapies for Duchenne muscular dystrophy and spinal muscular atrophy
- (2020) Mohsan Iftikhar et al. PHARMACOLOGY & THERAPEUTICS
- An Open‐Label, Multicenter, Phase I, Dose Escalation Study with Phase II Expansion Cohort to Determine the Safety, Pharmacokinetics, and Preliminary Antitumor Activity of Intravenous TKM‐080301 in Subjects with Advanced Hepatocellular Carcinoma
- (2019) Imane El Dika et al. ONCOLOGIST
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- (2019) Xuling Zhu et al. AMERICAN JOURNAL OF HUMAN GENETICS
- Emerging PEGylated non-biologic drugs
- (2019) Eun Ji Park et al. EXPERT OPINION ON EMERGING DRUGS
- Olaptesed pegol (NOX-A12) with bendamustine and rituximab: a phase IIa study in patients with relapsed/refractory chronic lymphocytic leukemia
- (2019) Michael Steurer et al. HAEMATOLOGICA
- mRNA Therapy Improves Metabolic and Behavioral Abnormalities in a Murine Model of Citrin Deficiency
- (2019) Jingsong Cao et al. MOLECULAR THERAPY
- The Potential for microRNA Therapeutics and Clinical Research
- (2019) Johora Hanna et al. Frontiers in Genetics
- Gene Therapy for Methylmalonic Acidemia: Past, Present, and Future
- (2019) Randy Joseph Chandler et al. HUMAN GENE THERAPY
- Advances in gene therapy for cystic fibrosis lung disease
- (2019) Ziying Yan et al. HUMAN MOLECULAR GENETICS
- miR-155 as a novel clinical target for hematological malignancies
- (2019) Lisa Witten et al. CARCINOGENESIS
- miR‐15a‐5p, miR‐15b‐5p, and miR‐16‐5p inhibit tumor progression by directly targeting MYCN in neuroblastoma
- (2019) Srinivas Chava et al. Molecular Oncology
- Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease
- (2019) Jinkuk Kim et al. NEW ENGLAND JOURNAL OF MEDICINE
- Neuropathy Associated with Systemic Amyloidosis
- (2019) Michelle Kaku et al. SEMINARS IN NEUROLOGY
- Discovery and preclinical evaluation of anti-miR-17 oligonucleotide RGLS4326 for the treatment of polycystic kidney disease
- (2019) Edmund C. Lee et al. Nature Communications
- Leading RNA Interference Therapeutics Part 2: Silencing Delta-Aminolevulinic Acid Synthase 1, with a Focus on Givosiran
- (2019) Pedro Renato de Paula Brandão et al. Molecular Diagnosis & Therapy
- The AGO proteins: an overview
- (2018) Saife Niaz BIOLOGICAL CHEMISTRY
- Final results from a defibrotide treatment-IND study for patients with hepatic veno-occlusive disease/sinusoidal obstruction syndrome
- (2018) Nancy A. Kernan et al. BRITISH JOURNAL OF HAEMATOLOGY
- The biology and role of CD44 in cancer progression: therapeutic implications
- (2018) Chen Chen et al. Journal of Hematology & Oncology
- Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis
- (2018) David Adams et al. NEW ENGLAND JOURNAL OF MEDICINE
- Inotersen Treatment for Patients with Hereditary Transthyretin Amyloidosis
- (2018) Merrill D. Benson et al. NEW ENGLAND JOURNAL OF MEDICINE
- Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy
- (2018) Eugenio Mercuri et al. NEW ENGLAND JOURNAL OF MEDICINE
- Overexpression of miR‑221 and miR‑222 in the cancer stroma is associated with malignant potential in colorectal cancer
- (2018) Michihisa Iida et al. ONCOLOGY REPORTS
- Multi-antigenic human cytomegalovirus mRNA vaccines that elicit potent humoral and cell-mediated immunity
- (2018) Shinu John et al. VACCINE
- Cobomarsen, an oligonucleotide inhibitor of miR-155, co-ordinately regulates multiple survival pathways to reduce cellular proliferation and survival in cutaneous T-cell lymphoma
- (2018) Anita G. Seto et al. BRITISH JOURNAL OF HAEMATOLOGY
- Patisiran: First Global Approval
- (2018) Sheridan M. Hoy DRUGS
- A synthetic microRNA-92a inhibitor (MRG-110) accelerates angiogenesis and wound healing in diabetic and non-diabetic wounds
- (2018) Corrie L. Gallant-Behm et al. WOUND REPAIR AND REGENERATION
- A microRNA-29 mimic (remlarsen) represses extracellular matrix expression and fibroplasia in the skin
- (2018) Corrie L. Gallant-Behm et al. JOURNAL OF INVESTIGATIVE DERMATOLOGY
- Safety and activity of microRNA-loaded minicells in patients with recurrent malignant pleural mesothelioma: a first-in-man, phase 1, open-label, dose-escalation study
- (2017) Nico van Zandwijk et al. LANCET ONCOLOGY
- miR-34a is downregulated in human osteosarcoma stem-like cells and promotes invasion, tumorigenic ability and self-renewal capacity
- (2017) Yonggen Zou et al. Molecular Medicine Reports
- Progress toward Gene Therapy for Duchenne Muscular Dystrophy
- (2017) Joel R. Chamberlain et al. MOLECULAR THERAPY
- Functional 5′ UTR mRNA structures in eukaryotic translation regulation and how to find them
- (2017) Kathrin Leppek et al. NATURE REVIEWS MOLECULAR CELL BIOLOGY
- Prognostic value of miRNA-155 expression in B-cell Non-Hodgkin's lymphoma
- (2017) Ahmed M. L. Bedewy et al. Turkish Journal of Hematology
- FDA Approval of Nusinersen for Spinal Muscular Atrophy Makes 2016 the Year of Splice Modulating Oligonucleotides
- (2017) Annemieke Aartsma-Rus Nucleic Acid Therapeutics
- FDA Approves Eteplirsen for Duchenne Muscular Dystrophy: The Next Chapter in the Eteplirsen Saga
- (2017) Annemieke Aartsma-Rus et al. Nucleic Acid Therapeutics
- Systemic Messenger RNA Therapy as a Treatment for Methylmalonic Acidemia
- (2017) Ding An et al. Cell Reports
- Increasing Tumor-Infiltrating T Cells through Inhibition of CXCL12 with NOX-A12 Synergizes with PD-1 Blockade
- (2017) Dirk Zboralski et al. Cancer Immunology Research
- A phase I dose-escalation study of apatorsen (OGX-427), an antisense inhibitor targeting heat shock protein 27 (Hsp27), in patients with castration-resistant prostate cancer and other advanced cancers
- (2016) K. N. Chi et al. ANNALS OF ONCOLOGY
- Phase 3 trial of defibrotide for the treatment of severe veno-occlusive disease and multi-organ failure
- (2016) P. G. Richardson et al. BLOOD
- Expression and promotor hypermethylation of miR-34a in the various histological subtypes of ovarian cancer
- (2016) Gabriel Schmid et al. BMC CANCER
- Precise and efficient siRNA design: a key point in competent gene silencing
- (2016) E Fakhr et al. CANCER GENE THERAPY
- Methylmalonic and propionic acidemias
- (2016) Jamie L. Fraser et al. CURRENT OPINION IN PEDIATRICS
- Phase I study of MRX34, a liposomal miR-34a mimic, administered twice weekly in patients with advanced solid tumors
- (2016) Muhammad S. Beg et al. INVESTIGATIONAL NEW DRUGS
- An Investigational RNAi Therapeutic Targeting Glycolate Oxidase Reduces Oxalate Production in Models of Primary Hyperoxaluria
- (2016) Abigail Liebow et al. JOURNAL OF THE AMERICAN SOCIETY OF NEPHROLOGY
- Translation efficiency of mRNAs is increased by antisense oligonucleotides targeting upstream open reading frames
- (2016) Xue-hai Liang et al. NATURE BIOTECHNOLOGY
- Aptamers as targeted therapeutics: current potential and challenges
- (2016) Jiehua Zhou et al. NATURE REVIEWS DRUG DISCOVERY
- C-C motif-ligand 2 inhibition with emapticap pegol (NOX-E36) in type 2 diabetic patients with albuminuria
- (2016) Jan Menne et al. NEPHROLOGY DIALYSIS TRANSPLANTATION
- Overexpression of miR-29b reduces collagen biosynthesis by inhibiting heat shock protein 47 during skin wound healing
- (2016) Yumin Zhu et al. Translational Research
- An mRNA Vaccine Encoding Rabies Virus Glycoprotein Induces Protection against Lethal Infection in Mice and Correlates of Protection in Adult and Newborn Pigs
- (2016) Margit Schnee et al. PLoS Neglected Tropical Diseases
- Antisense oligonucleotides in therapy for neurodegenerative disorders
- (2015) Melvin M. Evers et al. ADVANCED DRUG DELIVERY REVIEWS
- Mipomersen and lomitapide: Two new drugs for the treatment of homozygous familial hypercholesterolemia
- (2015) Ioanna Gouni-Berthold et al. ATHEROSCLEROSIS SUPPLEMENTS
- Generation 2.5 Antisense Oligonucleotides Targeting the Androgen Receptor and Its Splice Variants Suppress Enzalutamide-Resistant Prostate Cancer Cell Growth
- (2015) Y. Yamamoto et al. CLINICAL CANCER RESEARCH
- Mipomersen preferentially reduces small low-density lipoprotein particle number in patients with hypercholesterolemia
- (2015) Raul D. Santos et al. Journal of Clinical Lipidology
- Towards a molecular understanding of microRNA-mediated gene silencing
- (2015) Stefanie Jonas et al. NATURE REVIEWS GENETICS
- Crystal structure of a mirror-image L-RNA aptamer (Spiegelmer) in complex with the natural L-protein target CCL2
- (2015) Dominik Oberthür et al. Nature Communications
- RNAi therapy targeting KRAS in combination with chemotherapy for locally advanced pancreatic cancer patients
- (2015) Talia Golan et al. Oncotarget
- Phase I clinical trial of adoptive cellular immunotherapy with APN401 in patients with solid tumors
- (2015) Pierre Triozzi et al. Journal for ImmunoTherapy of Cancer
- MicroRNA mimicry blocks pulmonary fibrosis
- (2014) R. L. Montgomery et al. EMBO Molecular Medicine
- Anti–microRNA-21 oligonucleotides prevent Alport nephropathy progression by stimulating metabolic pathways
- (2014) Ivan G. Gomez et al. JOURNAL OF CLINICAL INVESTIGATION
- First-in-Human Phase I Study of the Liposomal RNA Interference Therapeutic Atu027 in Patients With Advanced Solid Tumors
- (2014) Beate Schultheis et al. JOURNAL OF CLINICAL ONCOLOGY
- The E3 ligase Cbl-b and TAM receptors regulate cancer metastasis via natural killer cells
- (2014) Magdalena Paolino et al. NATURE
- Nonsense-mediated decay as a terminating mechanism for antisense oligonucleotides
- (2014) Amanda J. Ward et al. NUCLEIC ACIDS RESEARCH
- Structural basis for microRNA targeting
- (2014) N. T. Schirle et al. SCIENCE
- Eteplirsen for the treatment of Duchenne muscular dystrophy
- (2013) Jerry R. Mendell et al. ANNALS OF NEUROLOGY
- Restoring expression of miR-16: a novel approach to therapy for malignant pleural mesothelioma
- (2013) G. Reid et al. ANNALS OF ONCOLOGY
- Treatment of HCV Infection by Targeting MicroRNA
- (2013) Harry L.A. Janssen et al. NEW ENGLAND JOURNAL OF MEDICINE
- Mutant KRAS is a druggable target for pancreatic cancer
- (2013) E. Zorde Khvalevsky et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Identification of Collagen 1 as a Post-transcriptional Target of miR-29b in Skin Fibroblasts: Therapeutic Implication for Scar Reduction
- (2012) Jie Cheng et al. AMERICAN JOURNAL OF THE MEDICAL SCIENCES
- VEGF gene therapy: therapeutic angiogenesis in the clinic and beyond
- (2012) M Giacca et al. GENE THERAPY
- Protective efficacy of in vitro synthesized, specific mRNA vaccines against influenza A virus infection
- (2012) Benjamin Petsch et al. NATURE BIOTECHNOLOGY
- MicroRNAs in metabolism and metabolic disorders
- (2012) Veerle Rottiers et al. NATURE REVIEWS MOLECULAR CELL BIOLOGY
- The mechanics of miRNA-mediated gene silencing: a look under the hood of miRISC
- (2012) Marc R Fabian et al. NATURE STRUCTURAL & MOLECULAR BIOLOGY
- The miRNA-212/132 family regulates both cardiac hypertrophy and cardiomyocyte autophagy
- (2012) Ahmet Ucar et al. Nature Communications
- Phase 2 randomized study of p53 antisense oligonucleotide (cenersen) plus idarubicin with or without cytarabine in refractory and relapsed acute myeloid leukemia
- (2011) Jorge Cortes et al. CANCER
- Phase I/II Trial of Custirsen (OGX-011), an Inhibitor of Clusterin, in Combination with a Gemcitabine and Platinum Regimen in Patients with Previously Untreated Advanced Non-small Cell Lung Cancer
- (2011) Janessa J. Laskin et al. Journal of Thoracic Oncology
- MicroRNAs 103 and 107 regulate insulin sensitivity
- (2011) Mirko Trajkovski et al. NATURE
- The microRNA miR-34a inhibits prostate cancer stem cells and metastasis by directly repressing CD44
- (2011) Can Liu et al. NATURE MEDICINE
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- (2010) Leah Cushing et al. AMERICAN JOURNAL OF RESPIRATORY CELL AND MOLECULAR BIOLOGY
- Gene signatures in hepatocellular carcinoma (HCC)
- (2010) Ourania M. Andrisani et al. SEMINARS IN CANCER BIOLOGY
- Phase II study of a TLR-9 agonist (1018 ISS) with rituximab in patients with relapsed or refractory follicular lymphoma
- (2009) Jonathan W. Friedberg et al. BRITISH JOURNAL OF HAEMATOLOGY
- miR-15a and miR-16 Are Implicated in Cell Cycle Regulation in a Rb-Dependent Manner and Are Frequently Deleted or Down-regulated in Non–Small Cell Lung Cancer
- (2009) Nora Bandi et al. CANCER RESEARCH
- MicroRNA miR-34 Inhibits Human Pancreatic Cancer Tumor-Initiating Cells
- (2009) Qing Ji et al. PLoS One
- MicroRNA-92a Controls Angiogenesis and Functional Recovery of Ischemic Tissues in Mice
- (2009) A. Bonauer et al. SCIENCE
- The miR-15a–miR-16-1 cluster controls prostate cancer by targeting multiple oncogenic activities
- (2008) Désirée Bonci et al. NATURE MEDICINE
- Age-Related Macular Degeneration
- (2008) Rama D. Jager et al. NEW ENGLAND JOURNAL OF MEDICINE
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