Tapping the RNA world for therapeutics

Functional Classification and Experimental Dissection of Long Noncoding RNAs

(2018) Florian Kopp et al.   CELL

A modular platform for targeted RNAi therapeutics

(2018) Ranit Kedmi et al.   Nature Nanotechnology

mRNA vaccines — a new era in vaccinology

(2018) Norbert Pardi et al.   NATURE REVIEWS DRUG DISCOVERY

Tisagenlecleucel in Children and Young Adults with B-Cell Lymphoblastic Leukemia

(2018) Shannon L. Maude et al.   NEW ENGLAND JOURNAL OF MEDICINE

Gene therapy comes of age

(2018) Cynthia E. Dunbar et al.   SCIENCE

Nucleic Acid Therapies for Cystic Fibrosis

(2018) Shruti Sasaki et al.   Nucleic Acid Therapeutics

Efficacy and safety of voretigene neparvovec (AAV2-hRPE65v2) in patients with RPE65 -mediated inherited retinal dystrophy: a randomised, controlled, open-label, phase 3 trial

(2017) Stephen Russell et al.   LANCET

Clinical Proof of Concept for a Novel Hepatocyte-Targeting GalNAc-siRNA Conjugate

(2017) Tracy S. Zimmermann et al.   MOLECULAR THERAPY

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

(2017) Norbert Pardi et al.   NATURE

Worth the RISC?

(2017) Ken Garber   NATURE BIOTECHNOLOGY

The chemical evolution of oligonucleotide therapies of clinical utility

(2017) Anastasia Khvorova et al.   NATURE BIOTECHNOLOGY

Cellular uptake and trafficking of antisense oligonucleotides

(2017) Stanley T Crooke et al.   NATURE BIOTECHNOLOGY

Overcoming cellular barriers for RNA therapeutics

(2017) Steven F Dowdy   NATURE BIOTECHNOLOGY

Refining strategies to translate genome editing to the clinic

(2017) Tatjana I Cornu et al.   NATURE MEDICINE

RNA-based recognition and targeting: sowing the seeds of specificity

(2017) Stanislaw A. Gorski et al.   NATURE REVIEWS MOLECULAR CELL BIOLOGY

Targeting of Antithrombin in Hemophilia A or B with RNAi Therapy

(2017) K. John Pasi et al.   NEW ENGLAND JOURNAL OF MEDICINE

A Highly Durable RNAi Therapeutic Inhibitor of PCSK9

(2017) Kevin Fitzgerald et al.   NEW ENGLAND JOURNAL OF MEDICINE

Axicabtagene Ciloleucel CAR T-Cell Therapy in Refractory Large B-Cell Lymphoma

(2017) Sattva S. Neelapu et al.   NEW ENGLAND JOURNAL OF MEDICINE

Nusinersen versus Sham Control in Infantile-Onset Spinal Muscular Atrophy

(2017) Richard S. Finkel et al.   NEW ENGLAND JOURNAL OF MEDICINE

Asialoglycoprotein receptor 1 mediates productive uptake of N-acetylgalactosamine-conjugated and unconjugated phosphorothioate antisense oligonucleotides into liver hepatocytes

(2017) Michael Tanowitz et al.   NUCLEIC ACIDS RESEARCH

The Dimensions, Dynamics, and Relevance of the Mammalian Noncoding Transcriptome

(2017) Ira W. Deveson et al.   TRENDS IN GENETICS

Molecular Mechanisms of Antisense Oligonucleotides

(2017) Stanley T. Crooke   Nucleic Acid Therapeutics

Effect of PCSK9 Inhibitors on Clinical Outcomes in Patients With Hypercholesterolemia: A Meta‐Analysis of 35 Randomized Controlled Trials

(2017) Aris Karatasakis et al.   Journal of the American Heart Association

Preclinical and Clinical Advances of GalNAc-Decorated Nucleic Acid Therapeutics

(2017) Yuanyu Huang   Molecular Therapy-Nucleic Acids

Seq and You Will Find

(2016) Nicole Schonrock et al.   CURRENT GENE THERAPY

Embracing proteins: structural themes in aptamer–protein complexes

(2016) Amy D Gelinas et al.   CURRENT OPINION IN STRUCTURAL BIOLOGY

Advances in RNAi therapeutic delivery to leukocytes using lipid nanoparticles

(2016) Srinivas Ramishetti et al.   JOURNAL OF DRUG TARGETING

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

Inhibition of Glycolate Oxidase With Dicer-substrate siRNA Reduces Calcium Oxalate Deposition in a Mouse Model of Primary Hyperoxaluria Type 1

(2016) Chaitali Dutta et al.   MOLECULAR THERAPY

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

(2016) Lena M. Kranz et al.   NATURE

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

(2016) Xue-hai Liang et al.   NATURE BIOTECHNOLOGY

Cornerstones of CRISPR–Cas in drug discovery and therapy

(2016) Christof Fellmann et al.   NATURE REVIEWS DRUG DISCOVERY

Aptamers as targeted therapeutics: current potential and challenges

(2016) Jiehua Zhou et al.   NATURE REVIEWS DRUG DISCOVERY

Unique features of long non-coding RNA biogenesis and function

(2016) Jeffrey J. Quinn et al.   NATURE REVIEWS GENETICS

Docosahexaenoic Acid Conjugation Enhances Distribution and Safety of siRNA upon Local Administration in Mouse Brain

(2016) Mehran Nikan et al.   Molecular Therapy-Nucleic Acids

siRNA Conjugates Carrying Sequentially Assembled Trivalent N-Acetylgalactosamine Linked Through Nucleosides Elicit Robust Gene Silencing In Vivo in Hepatocytes

(2015) Shigeo Matsuda et al.   ACS Chemical Biology

PIWI-Interacting RNA: Its Biogenesis and Functions

(2015) Yuka W. Iwasaki et al.   Annual Review of Biochemistry

Hepatocyte-Specific Delivery of siRNAs Conjugated to Novel Non-nucleosidic TrivalentN-Acetylgalactosamine Elicits Robust Gene Silencing in Vivo

(2015) Kallanthottathil G. Rajeev et al.   CHEMBIOCHEM

Manipulating the in vivo immune response by targeted gene knockdown

(2015) Judy Lieberman   CURRENT OPINION IN IMMUNOLOGY

The roles of CRISPR–Cas systems in adaptive immunity and beyond

(2015) Rodolphe Barrangou   CURRENT OPINION IN IMMUNOLOGY

Gene Knockdown by EpCAM Aptamer-siRNA Chimeras Suppresses Epithelial Breast Cancers and Their Tumor-Initiating Cells

(2015) A. Gilboa-Geffen et al.   MOLECULAR CANCER THERAPEUTICS

mRNA: Fulfilling the Promise of Gene Therapy

(2015) Drew Weissman et al.   MOLECULAR THERAPY

Visualizing lipid-formulated siRNA release from endosomes and target gene knockdown

(2015) Anders Wittrup et al.   NATURE BIOTECHNOLOGY

Knocking down disease: a progress report on siRNA therapeutics

(2015) Anders Wittrup et al.   NATURE REVIEWS GENETICS

RNA-mediated epigenetic regulation of gene expression

(2015) Daniel Holoch et al.   NATURE REVIEWS GENETICS

Harnessing RNAi-based nanomedicines for therapeutic gene silencing in B-cell malignancies

(2015) Shiri Weinstein et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

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

(2015) M. Tabebordbar et al.   SCIENCE

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

(2015) C. E. Nelson et al.   SCIENCE

The Noncoding RNA Revolution—Trashing Old Rules to Forge New Ones

(2014) Thomas R. Cech et al.   CELL

Multivalent N-Acetylgalactosamine-Conjugated siRNA Localizes in Hepatocytes and Elicits Robust RNAi-Mediated Gene Silencing

(2014) Jayaprakash K. Nair et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

Posttranscriptional Regulation of Gene Expression by Piwi Proteins and piRNAs

(2014) Toshiaki Watanabe et al.   MOLECULAR CELL

Multifunctional Aptamer-miRNA Conjugates for Targeted Cancer Therapy

(2014) Carla L Esposito et al.   MOLECULAR THERAPY

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

(2014) Hao Yin et al.   NATURE BIOTECHNOLOGY

Targeted delivery of antisense oligonucleotides to hepatocytes using triantennaryN-acetyl galactosamine improves potency 10-fold in mice

(2014) Thazha P. Prakash et al.   NUCLEIC ACIDS RESEARCH

RNAi-mediated silencing of hepatic Alas1 effectively prevents and treats the induced acute attacks in acute intermittent porphyria mice

(2014) M. Yasuda et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Beyond antibodies: New affinity reagents to unlock the proteome

(2014) Bridget Lollo et al.   PROTEOMICS

A Decade of Riboswitches

(2013) Alexander Serganov et al.   CELL

Image-based analysis of lipid nanoparticle–mediated siRNA delivery, intracellular trafficking and endosomal escape

(2013) Jerome Gilleron et al.   NATURE BIOTECHNOLOGY

Treatment of HCV Infection by Targeting MicroRNA

(2013) Harry L.A. Janssen et al.   NEW ENGLAND JOURNAL OF MEDICINE

Roles for MicroRNAs in Conferring Robustness to Biological Processes

(2012) Margaret S. Ebert et al.   CELL

RNA therapeutics: beyond RNA interference and antisense oligonucleotides

(2012) Ryszard Kole et al.   NATURE REVIEWS DRUG DISCOVERY

Structure-based druggability assessment—identifying suitable targets for small molecule therapeutics

(2011) Eric B Fauman et al.   CURRENT OPINION IN CHEMICAL BIOLOGY

Exon skipping and dystrophin restoration in patients with Duchenne muscular dystrophy after systemic phosphorodiamidate morpholino oligomer treatment: an open-label, phase 2, dose-escalation study

(2011) Sebahattin Cirak et al.   LANCET

CRISPR RNA maturation by trans-encoded small RNA and host factor RNase III

(2011) Elitza Deltcheva et al.   NATURE

Advances in human proteomics at high scale with the SOMAscan proteomics platform

(2011) Larry Gold et al.   New Biotechnology

Highly Efficient Reprogramming to Pluripotency and Directed Differentiation of Human Cells with Synthetic Modified mRNA

(2010) Luigi Warren et al.   Cell Stem Cell

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

(2010) Frederick J Raal et al.   LANCET

Aptamers as therapeutics

(2010) Anthony D. Keefe et al.   NATURE REVIEWS DRUG DISCOVERY

Desperately seeking microRNA targets

(2010) Marshall Thomas et al.   NATURE STRUCTURAL & MOLECULAR BIOLOGY

Riboswitches and the RNA World

(2010) R. R. Breaker   Cold Spring Harbor Perspectives in Biology

A Genome-wide RNAi Screen Identifies Multiple Synthetic Lethal Interactions with the Ras Oncogene

(2009) Ji Luo et al.   CELL

MicroRNAs: Target Recognition and Regulatory Functions

(2009) David P. Bartel   CELL

Identifying druggable disease-modifying gene products

(2009) Scott J Dixon et al.   CURRENT OPINION IN CHEMICAL BIOLOGY

In vivo delivery of siRNA to immune cells by conjugation to a TLR9 agonist enhances antitumor immune responses

(2009) Marcin Kortylewski et al.   NATURE BIOTECHNOLOGY

Systemic administration of optimized aptamer-siRNA chimeras promotes regression of PSMA-expressing tumors

(2009) Justin P Dassie et al.   NATURE BIOTECHNOLOGY

Incorporation of Pseudouridine Into mRNA Yields Superior Nonimmunogenic Vector With Increased Translational Capacity and Biological Stability

(2008) Katalin Karikó et al.   MOLECULAR THERAPY

Systemic Leukocyte-Directed siRNA Delivery Revealing Cyclin D1 as an Anti-Inflammatory Target

(2008) D. Peer et al.   SCIENCE

Cancer Proliferation Gene Discovery Through Functional Genomics

(2008) M. R. Schlabach et al.   SCIENCE

Profiling Essential Genes in Human Mammary Cells by Multiplex RNAi Screening

(2008) J. M. Silva et al.   SCIENCE

CRISPR Interference Limits Horizontal Gene Transfer in Staphylococci by Targeting DNA

(2008) Luciano A. Marraffini et al.   SCIENCE

Identification of Host Proteins Required for HIV Infection Through a Functional Genomic Screen

(2008) Abraham L. Brass et al.   SCIENCE