mRNA-based therapeutics — developing a new class of drugs

Generation of Gene-Modified Cynomolgus Monkey via Cas9/RNA-Mediated Gene Targeting in One-Cell Embryos

(2014) Yuyu Niu et al.   CELL

Effective gene targeting in rabbits using RNA-guided Cas9 nucleases

(2014) Dongshan Yang et al.   Journal of Molecular Cell Biology

A phase IB study on intravenous synthetic mRNA electroporated dendritic cell immunotherapy in pretreated advanced melanoma patients

(2013) S. Wilgenhof et al.   ANNALS OF ONCOLOGY

mRNA-engineered mesenchymal stem cells for targeted delivery of interleukin-10 to sites of inflammation

(2013) O. Levy et al.   BLOOD

Exogenous mRNA delivery and bioavailability in gene transfer mediated by piggyBac transposition

(2013) Solenne Bire et al.   BMC BIOTECHNOLOGY

One-Step Generation of Mice Carrying Mutations in Multiple Genes by CRISPR/Cas-Mediated Genome Engineering

(2013) Haoyi Wang et al.   CELL

Generating rats with conditional alleles using CRISPR/Cas9

(2013) Yuanwu Ma et al.   CELL RESEARCH

Generation of gene-modified mice via Cas9/RNA-mediated gene targeting

(2013) Bin Shen et al.   CELL RESEARCH

Human iPSC-Based Modeling of Late-Onset Disease via Progerin-Induced Aging

(2013) Justine D. Miller et al.   Cell Stem Cell

Efficient Generation of Human iPSCs by a Synthetic Self-Replicative RNA

(2013) Naohisa Yoshioka et al.   Cell Stem Cell

Steps Toward Safe Cell Therapy Using Induced Pluripotent Stem Cells

(2013) Hideyuki Okano et al.   CIRCULATION RESEARCH

Eukaryotic translation initiation is controlled by cooperativity effects within ternary complexes of 4E-BP1, eIF4E, and the mRNA 5′ cap

(2013) Anna Modrak-Wojcik et al.   FEBS LETTERS

Regimen-Specific Effects of RNA-Modified Chimeric Antigen Receptor T Cells in Mice with Advanced Leukemia

(2013) David M. Barrett et al.   HUMAN GENE THERAPY

Modified Foxp3 mRNA protects against asthma through an IL-10–dependent mechanism

(2013) Lauren E. Mays et al.   JOURNAL OF CLINICAL INVESTIGATION

The regulatory landscape for actively personalized cancer immunotherapies

(2013) Cedrik M Britten et al.   NATURE BIOTECHNOLOGY

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

(2013) Lior Zangi et al.   NATURE BIOTECHNOLOGY

Efficient genome editing in zebrafish using a CRISPR-Cas system

(2013) Woong Y Hwang et al.   NATURE BIOTECHNOLOGY

High-frequency off-target mutagenesis induced by CRISPR-Cas nucleases in human cells

(2013) Yanfang Fu et al.   NATURE BIOTECHNOLOGY

Reprogramming human fibroblasts to pluripotency using modified mRNA

(2013) Pankaj K Mandal et al.   Nature Protocols

Generation of targeted mouse mutants by embryo microinjection of TALEN mRNA

(2013) Benedikt Wefers et al.   Nature Protocols

Safety and Efficacy of RNAi Therapy for Transthyretin Amyloidosis

(2013) Teresa Coelho et al.   NEW ENGLAND JOURNAL OF MEDICINE

In Vivo Messenger RNA Introduction into the Central Nervous System Using Polyplex Nanomicelle

(2013) Satoshi Uchida et al.   PLoS One

Site Specific Mutation of the Zic2 Locus by Microinjection of TALEN mRNA in Mouse CD1, C3H and C57BL/6J Oocytes

(2013) Benjamin Davies et al.   PLoS One

RNA-Guided Human Genome Engineering via Cas9

(2013) P. Mali et al.   SCIENCE

Synthetic Generation of Influenza Vaccine Viruses for Rapid Response to Pandemics

(2013) P. R. Dormitzer et al.   Science Translational Medicine

RNA: The new revolution in nucleic acid vaccines

(2013) Andrew J. Geall et al.   SEMINARS IN IMMUNOLOGY

Human induced pluripotent stem cells and their use in drug discovery for toxicity testing

(2013) Clay W Scott et al.   TOXICOLOGY LETTERS

RNA-Based Tools for Nuclear Reprogramming and Lineage-Conversion: Towards Clinical Applications

(2013) Juan A. Bernal   Journal of Cardiovascular Translational Research

mRNA

(2013) Sandra Van Lint et al.   Human Vaccines & Immunotherapeutics

Successful Use of mRNA‐Nucleofection for Overexpression of Interleukin‐10 in Murine Monocytes/Macrophages for Anti‐inflammatory Therapy in a Murine Model of Autoimmune Myocarditis

(2013) Oliver Zimmermann et al.   Journal of the American Heart Association

mTOR Inhibition Improves Antitumor Effects of Vaccination with Antigen-Encoding RNA

(2013) M. Diken et al.   Cancer Immunology Research

T Cells Expressing Chimeric Antigen Receptors Can Cause Anaphylaxis in Humans

(2013) M. V. Maus et al.   Cancer Immunology Research

Rapidly produced SAM® vaccine against H7N9 influenza is immunogenic in mice

(2013) Armin Hekele et al.   Emerging Microbes & Infections

Ways and means of eukaryotic mRNA decay

(2012) Vidya Balagopal et al.   Biochimica et Biophysica Acta-Gene Regulatory Mechanisms

Exploiting the Mutanome for Tumor Vaccination

(2012) J. C. Castle et al.   CANCER RESEARCH

Preclinical Evaluation of TriMix and Antigen mRNA-Based Antitumor Therapy

(2012) S. Van Lint et al.   CANCER RESEARCH

Vaccination with mRNA-Electroporated Dendritic Cells Induces Robust Tumor Antigen-Specific CD4+ and CD8+ T Cells Responses in Stage III and IV Melanoma Patients

(2012) E. H. J. G. Aarntzen et al.   CLINICAL CANCER RESEARCH

mRNA as a Versatile Tool for Exogenous Protein Expression

(2012) Andreas N. Kuhn et al.   CURRENT GENE THERAPY

Insertional Mutagenesis by a Hybrid PiggyBac and Sleeping Beauty Transposon in the Rat

(2012) Kenryo Furushima et al.   GENETICS

Highly potent mRNA based cancer vaccines represent an attractive platform for combination therapies supporting an improved therapeutic effect

(2012) Mariola Fotin-Mleczek et al.   JOURNAL OF GENE MEDICINE

B Cell TLR7 Expression Drives Anti-RNA Autoantibody Production and Exacerbates Disease in Systemic Lupus Erythematosus-Prone Mice

(2012) S.-H. Hwang et al.   JOURNAL OF IMMUNOLOGY

Dendritic Cells Loaded With mRNA Encoding Full-length Tumor Antigens Prime CD4+ and CD8+ T Cells in Melanoma Patients

(2012) An MT Van Nuffel et al.   MOLECULAR THERAPY

Increased Erythropoiesis in Mice Injected With Submicrogram Quantities of Pseudouridine-containing mRNA Encoding Erythropoietin

(2012) Katalin Karikó et al.   MOLECULAR THERAPY

Systemic Delivery of Modified mRNA Encoding Herpes Simplex Virus 1 Thymidine Kinase for Targeted Cancer Gene Therapy

(2012) Yuhua Wang et al.   MOLECULAR THERAPY

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

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

(2012) Benjamin Petsch et al.   NATURE BIOTECHNOLOGY

Nonviral delivery of self-amplifying RNA vaccines

(2012) A. J. Geall et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Engineering genes for predictable protein expression

(2012) Claes Gustafsson et al.   PROTEIN EXPRESSION AND PURIFICATION

RNA-based vaccines

(2012) Jeffrey B. Ulmer et al.   VACCINE

Feeder-Free Derivation of Human Induced Pluripotent Stem Cells with Messenger RNA

(2012) Luigi Warren et al.   Scientific Reports

Targeting the tumor mutanome for personalized vaccination therapy

(2012) Sebastian Kreiter et al.   OncoImmunology

mRNA-based dendritic cell vaccination induces potent antiviral T-cell responses in HIV-1-infected patients

(2011) Ellen Van Gulck et al.   AIDS

Intravenous and intradermal TriMix-dendritic cell therapy results in a broad T-cell response and durable tumor response in a chemorefractory stage IV-M1c melanoma patient

(2011) An M. T. Van Nuffel et al.   CANCER IMMUNOLOGY IMMUNOTHERAPY

FLT3 Ligand Enhances the Cancer Therapeutic Potency of Naked RNA Vaccines

(2011) S. Kreiter et al.   CANCER RESEARCH

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

(2011) Sabine D. Allard et al.   CLINICAL IMMUNOLOGY

Nanoparticle-Derived Non-Viral Genetic Transfection at the Blood-Brain Barrier to Enable Neuronal Growth Factor Delivery by Secretion from Brain Endothelium

(2011) L. B. Thomsen et al.   CURRENT MEDICINAL CHEMISTRY

Tumor vaccination using messenger RNA: prospects of a future therapy

(2011) Sebastian Kreiter et al.   CURRENT OPINION IN IMMUNOLOGY

Selective uptake of naked vaccine RNA by dendritic cells is driven by macropinocytosis and abrogated upon DC maturation

(2011) M Diken et al.   GENE THERAPY

Treatment of Advanced Leukemia in Mice with mRNA Engineered T Cells

(2011) David M. Barrett et al.   HUMAN GENE THERAPY

Design of multifunctional non-viral gene vectors to overcome physiological barriers: Dilemmas and strategies

(2011) Tao Wang et al.   INTERNATIONAL JOURNAL OF PHARMACEUTICS

Therapeutic Vaccination With an Autologous mRNA Electroporated Dendritic Cell Vaccine in Patients With Advanced Melanoma

(2011) Sofie Wilgenhof et al.   JOURNAL OF IMMUNOTHERAPY

Detection of prokaryotic mRNA signifies microbial viability and promotes immunity

(2011) Leif E. Sander et al.   NATURE

Genetic engineering of human pluripotent cells using TALE nucleases

(2011) Dirk Hockemeyer et al.   NATURE BIOTECHNOLOGY

Is silence still golden? Mapping the RNAi patent landscape

(2011) Per Lundin   NATURE BIOTECHNOLOGY

Knockout rats generated by embryo microinjection of TALENs

(2011) Laurent Tesson et al.   NATURE BIOTECHNOLOGY

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

(2011) Michael S D Kormann et al.   NATURE BIOTECHNOLOGY

Ribose 2′-O-methylation provides a molecular signature for the distinction of self and non-self mRNA dependent on the RNA sensor Mda5

(2011) Roland Züst et al.   NATURE IMMUNOLOGY

Transfection of cells using flow-through electroporation based on constant voltage

(2011) Tao Geng et al.   Nature Protocols

RNAi-based therapeutic strategies for metabolic disease

(2011) Michael P. Czech et al.   Nature Reviews Endocrinology

Generating the optimal mRNA for therapy: HPLC purification eliminates immune activation and improves translation of nucleoside-modified, protein-encoding mRNA

(2011) Katalin Karikó et al.   NUCLEIC ACIDS RESEARCH

Nucleoside modifications in RNA limit activation of 2'-5'-oligoadenylate synthetase and increase resistance to cleavage by RNase L

(2011) B. R. Anderson et al.   NUCLEIC ACIDS RESEARCH

Protein expression from exogenous mRNA: Uptake by receptor-mediated endocytosis and trafficking via the lysosomal pathway

(2011) Christina Lorenz et al.   RNA Biology

Targeted Genome Editing Across Species Using ZFNs and TALENs

(2011) Andrew J. Wood et al.   SCIENCE

Tumor delivery of macromolecular drugs based on the EPR effect

(2010) Vladimir Torchilin   ADVANCED DRUG DELIVERY REVIEWS

From Allergen Genes to Allergy Vaccines

(2010) Rudolf Valenta et al.   Annual Review of Immunology

Reprogramming of human fibroblasts to pluripotent stem cells using mRNA of four transcription factors

(2010) Eduard Yakubov et al.   BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS

Particle size and activation threshold: a new dimension of danger signaling

(2010) L. Rettig et al.   BLOOD

Intranasal vaccination with messenger RNA as a new approach in gene therapy: Use against tuberculosis

(2010) Julio CC Lorenzi et al.   BMC BIOTECHNOLOGY

Intranodal Vaccination with Naked Antigen-Encoding RNA Elicits Potent Prophylactic and Therapeutic Antitumoral Immunity

(2010) S. Kreiter et al.   CANCER RESEARCH

Multiple Injections of Electroporated Autologous T Cells Expressing a Chimeric Antigen Receptor Mediate Regression of Human Disseminated Tumor

(2010) Y. Zhao et al.   CANCER RESEARCH

A Role for Codon Order in Translation Dynamics

(2010) Gina Cannarozzi et al.   CELL

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

(2010) Luigi Warren et al.   Cell Stem Cell

Prophylactic mRNA vaccination against allergy

(2010) Richard Weiss et al.   Current Opinion in Allergy and Clinical Immunology

Transiently redirected T cells for adoptive transfer

(2010) Hilde Almåsbak et al.   CYTOTHERAPY

Phosphorothioate cap analogs increase stability and translational efficiency of RNA vaccines in immature dendritic cells and induce superior immune responses in vivo

(2010) A N Kuhn et al.   GENE THERAPY

A simple and highly efficient transgenesis method in mice with the Tol2 transposon system and cytoplasmic microinjection

(2010) Kenta Sumiyama et al.   GENOMICS

Messenger RNA-based Vaccines With Dual Activity Induce Balanced TLR-7 Dependent Adaptive Immune Responses and Provide Antitumor Activity

(2010) Mariola Fotin-Mleczek et al.   JOURNAL OF IMMUNOTHERAPY

A Short Pulse of IL-4 Delivered by DCs Electroporated With Modified mRNA Can Both Prevent and Treat Autoimmune Diabetes in NOD Mice

(2010) Rémi J Creusot et al.   MOLECULAR THERAPY

Intradermal Vaccinations With RNA Coding for TAA Generate CD8+ and CD4+ Immune Responses and Induce Clinical Benefit in Vaccinated Patients

(2010) Susanne M Rittig et al.   MOLECULAR THERAPY

Rational design of cationic lipids for siRNA delivery

(2010) Sean C Semple et al.   NATURE BIOTECHNOLOGY

Incorporation of pseudouridine into mRNA enhances translation by diminishing PKR activation

(2010) Bart R. Anderson et al.   NUCLEIC ACIDS RESEARCH

Innate Immune Suppression Enables Frequent Transfection with RNA Encoding Reprogramming Proteins

(2010) Matthew Angel et al.   PLoS One

Induction of complete and molecular remissions in acute myeloid leukemia by Wilms' tumor 1 antigen-targeted dendritic cell vaccination

(2010) V. F. Van Tendeloo et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Novel endoribonucleases as central players in various pathways of eukaryotic RNA metabolism

(2010) R. Tomecki et al.   RNA

Regulation of mRNA decapping

(2010) You Li et al.   Wiley Interdisciplinary Reviews-RNA

Transposon-mediated BAC transgenesis in zebrafish and mice

(2009) Maximiliano L Suster et al.   BMC GENOMICS

The Many Pathways of RNA Degradation

(2009) Jonathan Houseley et al.   CELL

Single-Step Antigen Loading and Activation of Dendritic Cells by mRNA Electroporation for the Purpose of Therapeutic Vaccination in Melanoma Patients

(2009) A. Bonehill et al.   CLINICAL CANCER RESEARCH

Immunologic activity and safety of autologous HIV RNA-electroporated dendritic cells in HIV-1 infected patients receiving antiretroviral therapy

(2009) Jean-Pierre Routy et al.   CLINICAL IMMUNOLOGY

Clinical-grade manufacturing of autologous mature mRNA-electroporated dendritic cells and safety testing in acute myeloid leukemia patients in a phase I dose-escalation clinical trial

(2009) Ann Van Driessche et al.   CYTOTHERAPY

Endoribonucleases - enzymes gaining spotlight in mRNA metabolism

(2009) Wai Ming Li et al.   FEBS Journal

Chimeric Receptor mRNA Transfection as a Tool to Generate Antineoplastic Lymphocytes

(2009) Peter M. Rabinovich et al.   HUMAN GENE THERAPY

Recognition of 5′ Triphosphate by RIG-I Helicase Requires Short Blunt Double-Stranded RNA as Contained in Panhandle of Negative-Strand Virus

(2009) Martin Schlee et al.   IMMUNITY

Immunize and disappear—Safety-optimized mRNA vaccination with a panel of 29 allergens

(2009) Elisabeth Roesler et al.   JOURNAL OF ALLERGY AND CLINICAL IMMUNOLOGY

Direct Injection of Protamine-protected mRNA: Results of a Phase 1/2 Vaccination Trial in Metastatic Melanoma Patients

(2009) Benjamin Weide et al.   JOURNAL OF IMMUNOTHERAPY

Activation of MDA5 Requires Higher-Order RNA Structures Generated during Virus Infection

(2009) A. Pichlmair et al.   JOURNAL OF VIROLOGY

RNA stability: is it the endo' the world as we know it?

(2009) Jeffrey Wilusz   NATURE STRUCTURAL & MOLECULAR BIOLOGY

Knockout Rats via Embryo Microinjection of Zinc-Finger Nucleases

(2009) Aron M. Geurts et al.   SCIENCE

Alphaviruses in Gene Therapy

(2009) Kenneth Lundstrom   Viruses-Basel

Targeted delivery of nanoparticles for the treatment of lung diseases

(2008) Shirzad Azarmi et al.   ADVANCED DRUG DELIVERY REVIEWS

Adoptive immunotherapy using human peripheral blood lymphocytes transferred with RNA encoding Her-2/neu-specific chimeric immune receptor in ovarian cancer xenograft model

(2008) S H Yoon et al.   CANCER GENE THERAPY

DNA vaccines for the prevention and treatment of allergy

(2008) Kaw Yan Chua et al.   Current Opinion in Allergy and Clinical Immunology

Messenger RNA regulation: to translate or to degrade

(2008) Ann-Bin Shyu et al.   EMBO JOURNAL

Selective Modification of Antigen-Specific T Cells by RNA Electroporation

(2008) Duane A. Mitchell et al.   HUMAN GENE THERAPY

Bax mRNA therapy using cationic liposomes for human malignant melanoma

(2008) Kenya Okumura et al.   JOURNAL OF GENE MEDICINE

Recommendations on risk-based strategies for detection and characterization of antibodies against biotechnology products

(2008) Eugen Koren et al.   JOURNAL OF IMMUNOLOGICAL METHODS

Cytokine Maturation Followed by CD40L mRNA Electroporation Results in a Clinically Relevant Dendritic Cell Product Capable of Inducing a Potent Proinflammatory CTL Response

(2008) David M. Calderhead et al.   JOURNAL OF IMMUNOTHERAPY

Results of the First Phase I/II Clinical Vaccination Trial With Direct Injection of mRNA

(2008) Benjamin Weide et al.   JOURNAL OF IMMUNOTHERAPY

Enhancing the T-cell Stimulatory Capacity of Human Dendritic Cells by Co-electroporation With CD40L, CD70 and Constitutively Active TLR4 Encoding mRNA

(2008) Aude Bonehill et al.   MOLECULAR THERAPY

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

(2008) Katalin Karikó et al.   MOLECULAR THERAPY

Heritable targeted gene disruption in zebrafish using designed zinc-finger nucleases

(2008) Yannick Doyon et al.   NATURE BIOTECHNOLOGY

Synthesis and characterization of mRNA cap analogs containing phosphorothioate substitutions that bind tightly to eIF4E and are resistant to the decapping pyrophosphatase DcpS

(2008) J. Kowalska et al.   RNA

Nucleoside modifications modulate activation of the protein kinase PKR in an RNA structure-specific manner

(2008) S. R. Nallagatla et al.   RNA