标题
RNA-based therapeutics: an overview and prospectus
作者
关键词
-
出版物
Cell Death & Disease
Volume 13, Issue 7, Pages -
出版商
Springer Science and Business Media LLC
发表日期
2022-07-23
DOI
10.1038/s41419-022-05075-2
参考文献
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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。- Circular RNA vaccines against SARS-CoV-2 and emerging variants
- (2022) Liang Qu et al. CELL
- Lumasiran: First Approval
- (2021) Lesley J. Scott et al. DRUGS
- BNT162b2 mRNA COVID-19 Vaccine: First Approval
- (2021) Yvette N. Lamb DRUGS
- mRNA vaccine: a potential therapeutic strategy
- (2021) Yang Wang et al. Molecular Cancer
- mRNA vaccine for cancer immunotherapy
- (2021) Lei Miao et al. Molecular Cancer
- In Situ Transforming RNA Nanovaccines from Polyethylenimine Functionalized Graphene Oxide Hydrogel for Durable Cancer Immunotherapy
- (2021) Yue Yin et al. NANO LETTERS
- Engineered ionizable lipid nanoparticles for targeted delivery of RNA therapeutics into different types of cells in the liver
- (2021) M. Kim et al. Science Advances
- Casimersen: First Approval
- (2021) Matt Shirley DRUGS
- Clinical and immunological effects of mRNA vaccines in malignant diseases
- (2021) Annkristin Heine et al. Molecular Cancer
- mRNA therapeutics in cancer immunotherapy
- (2021) Jan D. Beck et al. Molecular Cancer
- Antisense technology: an overview and prospectus
- (2021) Stanley T. Crooke et al. NATURE REVIEWS DRUG DISCOVERY
- MTL-CEBPA Combined with Immunotherapy or RFA Enhances Immunological Anti-Tumor Response in Preclinical Models
- (2021) Kai-Wen Huang et al. INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
- mRNA vaccines for infectious diseases: principles, delivery and clinical translation
- (2021) Namit Chaudhary et al. NATURE REVIEWS DRUG DISCOVERY
- Noncoding RNA therapeutics — challenges and potential solutions
- (2021) Melanie Winkle et al. NATURE REVIEWS DRUG DISCOVERY
- The Importance of Apparent pKa in the Development of Nanoparticles Encapsulating siRNA and mRNA
- (2021) Pratikkumar Patel et al. TRENDS IN PHARMACOLOGICAL SCIENCES
- Principles for designing an optimal mRNA lipid nanoparticle vaccine
- (2021) Edo Kon et al. CURRENT OPINION IN BIOTECHNOLOGY
- RNA circles with minimized immunogenicity as potent PKR inhibitors
- (2021) Chu-Xiao Liu et al. MOLECULAR CELL
- RNA Activation—A Novel Approach to Therapeutically Upregulate Gene Transcription
- (2021) Choon Ping Tan et al. MOLECULES
- Golodirsen: First Approval
- (2020) Young-A Heo DRUGS
- Givosiran: First Approval
- (2020) Lesley J. Scott DRUGS
- The biology, function, and biomedical applications of exosomes
- (2020) Raghu Kalluri et al. SCIENCE
- CasRx-mediated RNA targeting prevents choroidal neovascularization in a mouse model of age-related macular degeneration
- (2020) Changyang Zhou et al. National Science Review
- MTL-CEBPA, a small activating RNA therapeutic up-regulating C/EBP-α, in patients with advanced liver cancer: a first-in-human, multi-centre, open-label, phase I trial
- (2020) Debashis Sarker et al. CLINICAL CANCER RESEARCH
- A Mechanism for microRNA Arm Switching Regulated by Uridylation
- (2020) Haedong Kim et al. MOLECULAR CELL
- Engineered Circular RNA Sponges Act as miRNA Inhibitors to Attenuate Pressure Overload-Induced Cardiac Hypertrophy
- (2020) Annadoray Lavenniah et al. MOLECULAR THERAPY
- Selective organ targeting (SORT) nanoparticles for tissue-specific mRNA delivery and CRISPR–Cas gene editing
- (2020) Qiang Cheng et al. Nature Nanotechnology
- RNA therapeutics on the rise
- (2020) Feng Wang et al. NATURE REVIEWS DRUG DISCOVERY
- Modulation of metabolic functions through Cas13d-mediated gene knockdown in liver
- (2020) Bingbing He et al. Protein & Cell
- Lipid nanoparticle technology for therapeutic gene regulation in the liver
- (2020) Dominik Witzigmann et al. ADVANCED DRUG DELIVERY REVIEWS
- Viltolarsen: First Approval
- (2020) Sohita Dhillon DRUGS
- A Potent Branched-Tail Lipid Nanoparticle Enables Multiplexed mRNA Delivery and Gene Editing In Vivo
- (2020) Khalid A. Hajj et al. NANO LETTERS
- SARS-CoV-2 mRNA vaccine design enabled by prototype pathogen preparedness
- (2020) Kizzmekia S. Corbett et al. NATURE
- Risdiplam: First Approval
- (2020) Sohita Dhillon DRUGS
- Recent advances in chemical modifications of guide RNA, mRNA and donor template for CRISPR-mediated genome editing
- (2020) Qiubing Chen et al. ADVANCED DRUG DELIVERY REVIEWS
- Non-coding RNAs and potential therapeutic targeting in cancer
- (2020) Shusuke Toden et al. BIOCHIMICA ET BIOPHYSICA ACTA-REVIEWS ON CANCER
- Efficacy and Safety of the mRNA-1273 SARS-CoV-2 Vaccine
- (2020) Lindsey R. Baden et al. NEW ENGLAND JOURNAL OF MEDICINE
- Development of a gene-editing approach to restore vision loss in Leber congenital amaurosis type 10
- (2019) Morgan L. Maeder et al. NATURE MEDICINE
- The current state and future directions of RNAi-based therapeutics
- (2019) Ryan L. Setten et al. NATURE REVIEWS DRUG DISCOVERY
- MicroRNA-34 family: a potential tumor suppressor and therapeutic candidate in cancer
- (2019) Lu Zhang et al. JOURNAL OF EXPERIMENTAL & CLINICAL CANCER RESEARCH
- Advances in CRISPR-Cas systems for RNA targeting, tracking and editing
- (2019) Fei Wang et al. BIOTECHNOLOGY ADVANCES
- The next generation of CRISPR–Cas technologies and applications
- (2019) Adrian Pickar-Oliver et al. NATURE REVIEWS MOLECULAR CELL BIOLOGY
- Using Large Datasets to Understand Nanotechnology
- (2019) Kalina Paunovska et al. ADVANCED MATERIALS
- Targeting mRNA processing as an anticancer strategy
- (2019) Joana Desterro et al. NATURE REVIEWS DRUG DISCOVERY
- Lomitapide and Mipomersen—Inhibiting Microsomal Triglyceride Transfer Protein (MTP) and apoB100 Synthesis
- (2019) Dirk J. Blom et al. Current Atherosclerosis Reports
- Aptamers: A Review of Their Chemical Properties and Modifications for Therapeutic Application
- (2019) Adachi et al. MOLECULES
- Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease
- (2019) Jinkuk Kim et al. NEW ENGLAND JOURNAL OF MEDICINE
- Applications of Spherical Nucleic Acid Nanoparticles as Delivery Systems
- (2019) Ahad Mokhtarzadeh et al. TRENDS IN MOLECULAR MEDICINE
- Spherical Nucleic Acid Nanoparticles: Therapeutic Potential
- (2018) Chintan H. Kapadia et al. BIODRUGS
- Emergence of synthetic mRNA: In vitro synthesis of mRNA and its applications in regenerative medicine
- (2018) Hyokyoung Kwon et al. BIOMATERIALS
- Transcriptome Engineering with RNA-Targeting Type VI-D CRISPR Effectors
- (2018) Silvana Konermann et al. CELL
- RNA-Targeted Therapeutics
- (2018) Stanley T. Crooke et al. Cell Metabolism
- CRISPR RNAs trigger innate immune responses in human cells
- (2018) Sojung Kim et al. GENOME RESEARCH
- Principles for targeting RNA with drug-like small molecules
- (2018) Katherine Deigan Warner et al. NATURE REVIEWS DRUG DISCOVERY
- 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
- Incorporation of bridged nucleic acids into CRISPR RNAs improves Cas9 endonuclease specificity
- (2018) Christopher R. Cromwell et al. Nature Communications
- RNA-dependent RNA targeting by CRISPR-Cas9
- (2018) Steven C Strutt et al. eLife
- Precision Medicine through Antisense Oligonucleotide-Mediated Exon Skipping
- (2018) Dunhui Li et al. TRENDS IN PHARMACOLOGICAL SCIENCES
- Development of MTL-CEBPA: Small Activating RNA Drug for Hepatocellular Carcinoma
- (2018) Ryan L. Setten et al. CURRENT PHARMACEUTICAL BIOTECHNOLOGY
- Novel upregulation of amyloid-β precursor protein (APP) by microRNA-346 via targeting of APP mRNA 5′-untranslated region: Implications in Alzheimer’s disease
- (2018) Justin M. Long et al. MOLECULAR PSYCHIATRY
- Chemical Modifications of Nucleic Acid Aptamers for Therapeutic Purposes
- (2017) Shuaijian Ni et al. INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES
- Clinical Proof of Concept for a Novel Hepatocyte-Targeting GalNAc-siRNA Conjugate
- (2017) Tracy S. Zimmermann et al. MOLECULAR THERAPY
- RNase H1-Dependent Antisense Oligonucleotides Are Robustly Active in Directing RNA Cleavage in Both the Cytoplasm and the Nucleus
- (2017) Xue-Hai Liang et al. MOLECULAR THERAPY
- The chemical evolution of oligonucleotide therapies of clinical utility
- (2017) Anastasia Khvorova et al. NATURE BIOTECHNOLOGY
- Overcoming cellular barriers for RNA therapeutics
- (2017) Steven F Dowdy NATURE BIOTECHNOLOGY
- Nusinersen, an antisense oligonucleotide drug for spinal muscular atrophy
- (2017) David R Corey NATURE NEUROSCIENCE
- Small molecules against RNA targets attract big backers
- (2017) Asher Mullard NATURE REVIEWS DRUG DISCOVERY
- MicroRNA therapeutics: towards a new era for the management of cancer and other diseases
- (2017) Rajesha Rupaimoole et al. NATURE REVIEWS DRUG DISCOVERY
- Impact of enhanced metabolic stability on pharmacokinetics and pharmacodynamics of GalNAc–siRNA conjugates
- (2017) Jayaprakash K. Nair et al. NUCLEIC ACIDS RESEARCH
- Improving CRISPR–Cas specificity with chemical modifications in single-guide RNAs
- (2017) Daniel E Ryan et al. NUCLEIC ACIDS RESEARCH
- Antisense oligonucleotides targeting translation inhibitory elements in 5′ UTRs can selectively increase protein levels
- (2017) Xue-hai Liang et al. NUCLEIC ACIDS RESEARCH
- The Race of 10 Synthetic RNAi-Based Drugs to the Pharmaceutical Market
- (2017) Ricardo Titze-de-Almeida et al. PHARMACEUTICAL RESEARCH
- RNA editing with CRISPR-Cas13
- (2017) David B. T. Cox et al. SCIENCE
- FDA Approval of Nusinersen for Spinal Muscular Atrophy Makes 2016 the Year of Splice Modulating Oligonucleotides
- (2017) Annemieke Aartsma-Rus Nucleic Acid Therapeutics
- The Clinical Application of MicroRNAs in Infectious Disease
- (2017) Ruth E. Drury et al. Frontiers in Immunology
- The role of helper lipids in lipid nanoparticles (LNPs) designed for oligonucleotide delivery
- (2016) Xinwei Cheng et al. ADVANCED DRUG DELIVERY REVIEWS
- saRNA-guided Ago2 targets the RITA complex to promoters to stimulate transcription
- (2016) Victoria Portnoy et al. CELL RESEARCH
- The Medicinal Chemistry of Therapeutic Oligonucleotides
- (2016) W. Brad Wan et al. JOURNAL OF MEDICINAL CHEMISTRY
- Polyethylenimine: A versatile, multifunctional non-viral vector for nucleic acid delivery
- (2016) Abhijeet P. Pandey et al. Materials Science & Engineering C-Materials for Biological Applications
- Genome-editing Technologies for Gene and Cell Therapy
- (2016) Morgan L Maeder et al. MOLECULAR THERAPY
- Alnylam terminates revusiran program, stock plunges
- (2016) Ken Garber NATURE BIOTECHNOLOGY
- Translation efficiency of mRNAs is increased by antisense oligonucleotides targeting upstream open reading frames
- (2016) Xue-hai Liang et al. NATURE BIOTECHNOLOGY
- Non-coding RNAs as drug targets
- (2016) Masayuki Matsui et al. NATURE REVIEWS DRUG DISCOVERY
- Aptamers as targeted therapeutics: current potential and challenges
- (2016) Jiehua Zhou et al. NATURE REVIEWS DRUG DISCOVERY
- Small activating RNA binds to the genomic target site in a seed-region-dependent manner
- (2016) Xing Meng et al. NUCLEIC ACIDS RESEARCH
- C2c2 is a single-component programmable RNA-guided RNA-targeting CRISPR effector
- (2016) Omar O. Abudayyeh et al. SCIENCE
- Biomaterials in siRNA Delivery: A Comprehensive Review
- (2016) William Ho et al. Advanced Healthcare Materials
- From selection hits to clinical leads: progress in aptamer discovery
- (2016) Keith E Maier et al. Molecular Therapy-Methods & Clinical Development
- Delivery of oligonucleotides with lipid nanoparticles
- (2015) Yuhua Wang et al. ADVANCED DRUG DELIVERY REVIEWS
- Preclinical and clinical development of siRNA-based therapeutics
- (2015) Gulnihal Ozcan et al. ADVANCED DRUG DELIVERY REVIEWS
- Self-Assembled DNA Nanoclews for the Efficient Delivery of CRISPR-Cas9 for Genome Editing
- (2015) Wujin Sun et al. ANGEWANDTE CHEMIE-INTERNATIONAL EDITION
- DNA nanotubes as intracellular delivery vehicles in vivo
- (2015) Sabine Sellner et al. BIOMATERIALS
- Treatment of neurological disorders by introducing mRNA in vivo using polyplex nanomicelles
- (2015) Miyuki Baba et al. JOURNAL OF CONTROLLED RELEASE
- Clinical Scale Zinc Finger Nuclease-mediated Gene Editing of PD-1 in Tumor Infiltrating Lymphocytes for the Treatment of Metastatic Melanoma
- (2015) Joal D Beane et al. MOLECULAR THERAPY
- Chemically modified guide RNAs enhance CRISPR-Cas genome editing in human primary cells
- (2015) Ayal Hendel et al. NATURE BIOTECHNOLOGY
- A general approach to high-yield biosynthesis of chimeric RNAs bearing various types of functional small RNAs for broad applications
- (2015) Qiu-Xia Chen et al. NUCLEIC ACIDS RESEARCH
- DNA Nanostructures as Smart Drug-Delivery Vehicles and Molecular Devices
- (2015) Veikko Linko et al. TRENDS IN BIOTECHNOLOGY
- The siRNA Non-seed Region and Its Target Sequences Are Auxiliary Determinants of Off-Target Effects
- (2015) Piotr J. Kamola et al. PLoS Computational Biology
- Locked nucleic acid/DNA chimeric aptamer probe for tumor diagnosis with improved serum stability and extended imaging window in vivo
- (2014) Hui Shi et al. ANALYTICA CHIMICA ACTA
- Aptamer-Targeted DNA Nanostructures for Therapeutic Delivery
- (2014) Phapanin Charoenphol et al. MOLECULAR PHARMACEUTICS
- Programmable RNA recognition and cleavage by CRISPR/Cas9
- (2014) Mitchell R. O’Connell et al. NATURE
- mRNA-based therapeutics — developing a new class of drugs
- (2014) Ugur Sahin et al. NATURE REVIEWS DRUG DISCOVERY
- Therapeutic targeting of microRNAs: current status and future challenges
- (2014) Zhonghan Li et al. NATURE REVIEWS DRUG DISCOVERY
- Nonsense-mediated decay as a terminating mechanism for antisense oligonucleotides
- (2014) Amanda J. Ward et al. NUCLEIC ACIDS RESEARCH
- The new frontier of genome engineering with CRISPR-Cas9
- (2014) J. A. Doudna et al. SCIENCE
- Phosphorothioates, Essential Components of Therapeutic Oligonucleotides
- (2014) Fritz Eckstein Nucleic Acid Therapeutics
- Cell-type-specific, Aptamer-functionalized Agents for Targeted Disease Therapy
- (2014) Jiehua Zhou et al. Molecular Therapy-Nucleic Acids
- Molecular Mechanisms of RNA Interference
- (2013) Ross C. Wilson et al. Annual Review of Biophysics
- Gene therapy on the move
- (2013) Kerstin B. Kaufmann et al. EMBO Molecular Medicine
- Influence of diverse chemical modifications on the ADME characteristics and toxicology of antisense oligonucleotides
- (2013) Mehrdad Dirin et al. EXPERT OPINION ON BIOLOGICAL THERAPY
- Natural RNA circles function as efficient microRNA sponges
- (2013) Thomas B. Hansen et al. NATURE
- Circular RNAs are a large class of animal RNAs with regulatory potency
- (2013) Sebastian Memczak et al. NATURE
- Image-based analysis of lipid nanoparticle–mediated siRNA delivery, intracellular trafficking and endosomal escape
- (2013) Jerome Gilleron et al. NATURE BIOTECHNOLOGY
- Mechanism for the endocytosis of spherical nucleic acid nanoparticle conjugates
- (2013) C. H. J. Choi et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- 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
- Spherical Nucleic Acid Nanoparticle Conjugates as an RNAi-Based Therapy for Glioblastoma
- (2013) S. A. Jensen et al. Science Translational Medicine
- Design and Development of Nanosized DNA Assemblies in Polypod-like Structures as Efficient Vehicles for Immunostimulatory CpG Motifs to Immune Cells
- (2012) Kohta Mohri et al. ACS Nano
- Exosomes for targeted siRNA delivery across biological barriers
- (2012) Samir EL Andaloussi et al. ADVANCED DRUG DELIVERY REVIEWS
- An open-label, phase 2 trial of RPI.4610 (angiozyme) in the treatment of metastatic breast cancer
- (2012) Phuong Khanh Morrow et al. CANCER
- Type I IFN Counteracts the Induction of Antigen-Specific Immune Responses by Lipid-Based Delivery of mRNA Vaccines
- (2012) Charlotte Pollard et al. MOLECULAR THERAPY
- Molecularly self-assembled nucleic acid nanoparticles for targeted in vivo siRNA delivery
- (2012) Hyukjin Lee et al. Nature Nanotechnology
- Development of Therapeutic-Grade Small Interfering RNAs by Chemical Engineering
- (2012) Jesper B. Bramsen et al. Frontiers in Genetics
- An Overview of Sugar-Modified Oligonucleotides for Antisense Therapeutics
- (2011) Thazha P. Prakash CHEMISTRY & BIODIVERSITY
- Exon-skipping therapy for Duchenne muscular dystrophy
- (2011) Akinori Nakamura et al. LANCET
- Challenges and opportunities for structural DNA nanotechnology
- (2011) Andre V. Pinheiro et al. Nature Nanotechnology
- Posttranscriptional Upregulation by MicroRNAs
- (2011) Shobha Vasudevan Wiley Interdisciplinary Reviews-RNA
- Therapeutic applications of ribozymes and riboswitches
- (2010) Jérôme Mulhbacher et al. CURRENT OPINION IN PHARMACOLOGY
- MicroRNA-466l Upregulates IL-10 Expression in TLR-Triggered Macrophages by Antagonizing RNA-Binding Protein Tristetraprolin-Mediated IL-10 mRNA Degradation
- (2010) F. Ma et al. JOURNAL OF IMMUNOLOGY
- Evidence of RNAi in humans from systemically administered siRNA via targeted nanoparticles
- (2010) Mark E. Davis et al. NATURE
- Chitosan-based formulations for delivery of DNA and siRNA
- (2009) Shirui Mao et al. ADVANCED DRUG DELIVERY REVIEWS
- Identifying druggable disease-modifying gene products
- (2009) Scott J Dixon et al. CURRENT OPINION IN CHEMICAL BIOLOGY
- The First Targeted Delivery of siRNA in Humans via a Self-Assembling, Cyclodextrin Polymer-Based Nanoparticle: From Concept to Clinic
- (2009) Mark E. Davis MOLECULAR PHARMACEUTICS
- The promises and pitfalls of RNA-interference-based therapeutics
- (2009) Daniela Castanotto et al. NATURE
- Phase 2 gene therapy trial of an anti-HIV ribozyme in autologous CD34+ cells
- (2009) Ronald T Mitsuyasu et al. NATURE MEDICINE
- Incorporation of Pseudouridine Into mRNA Yields Superior Nonimmunogenic Vector With Increased Translational Capacity and Biological Stability
- (2008) Katalin Karikó et al. MOLECULAR THERAPY
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