Immunological and hematological toxicities challenging clinical translation of nucleic acid-based therapeutics

Preclinical Evaluation of the Toxicological Effects of a Novel Constrained Ethyl Modified Antisense Compound Targeting Signal Transducer and Activator of Transcription 3 in Mice and Cynomolgus Monkeys

(2015) Sebastien A. Burel et al.   Nucleic Acid Therapeutics

Innate Immune Sensing and Signaling of Cytosolic Nucleic Acids

(2014) Jiaxi Wu et al.   Annual Review of Immunology

RNA:DNA hybrids are a novel molecular pattern sensed by TLR9

(2014) R. E. Rigby et al.   EMBO JOURNAL

A phase II trial of intraperitoneal EGEN-001, an IL-12 plasmid formulated with PEG–PEI–cholesterol lipopolymer in the treatment of persistent or recurrent epithelial ovarian, fallopian tube or primary peritoneal cancer: A Gynecologic Oncology Group study

(2014) Ronald D. Alvarez et al.   GYNECOLOGIC ONCOLOGY

Chemically Modified DNA Aptamers Bind Interleukin-6 with High Affinity and Inhibit Signaling by Blocking Its Interaction with Interleukin-6 Receptor

(2014) Shashi Gupta et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Synthetic immunostimulatory oligonucleotides in experimental and clinical practice

(2014) Paweł Bodera et al.   Pharmacological Reports

RNAi Therapies: Drugging the Undruggable

(2014) S. Y. Wu et al.   Science Translational Medicine

A phase 1 study of the BCL2-targeted deoxyribonucleic acid inhibitor (DNAi) PNT2258 in patients with advanced solid tumors

(2013) Anthony W. Tolcher et al.   CANCER CHEMOTHERAPY AND PHARMACOLOGY

Use of Oligonucleotide Aptamer Ligands to Modulate the Function of Immune Receptors

(2013) E. Gilboa et al.   CLINICAL CANCER RESEARCH

Clinical optimization of antigen specific modulation of type 1 diabetes with the plasmid DNA platform

(2013) Peter Gottlieb et al.   CLINICAL IMMUNOLOGY

Canonical and Non-Canonical Barriers Facing AntimiR Cancer Therapeutics

(2013) Christopher Cheng et al.   CURRENT MEDICINAL CHEMISTRY

Phase I trial of a formulated IL-12 plasmid in combination with carboplatin and docetaxel chemotherapy in the treatment of platinum-sensitive recurrent ovarian cancer

(2013) Khursheed Anwer et al.   GYNECOLOGIC ONCOLOGY

In vivo comparison of local versus systemic delivery of immunostimulating siRNA in HPV-driven tumours

(2013) Norliana Khairuddin et al.   IMMUNOLOGY AND CELL BIOLOGY

A phase II trial of AS1411 (a novel nucleolin-targeted DNA aptamer) in metastatic renal cell carcinoma

(2013) Jonathan E. Rosenberg et al.   INVESTIGATIONAL NEW DRUGS

Safety, Tolerability, and Immunogenicity of Repeated Doses of DermaVir, a Candidate Therapeutic HIV Vaccine, in HIV-Infected Patients Receiving Combination Antiretroviral Therapy

(2013) Benigno Rodriguez et al.   JAIDS-JOURNAL OF ACQUIRED IMMUNE DEFICIENCY SYNDROMES

Spermine-alt-poly(ethylene glycol) polyspermine as a safe and efficient aerosol gene carrier for lung cancer therapy

(2013) You-Kyoung Kim et al.   JOURNAL OF BIOMEDICAL MATERIALS RESEARCH PART A

Liver as a target for oligonucleotide therapeutics

(2013) Alfica Sehgal et al.   JOURNAL OF HEPATOLOGY

Aptamers: multifunctional molecules for biomedical research

(2013) Jayeeta Banerjee et al.   JOURNAL OF MOLECULAR MEDICINE-JMM

Delivery materials for siRNA therapeutics

(2013) Rosemary Kanasty et al.   NATURE MATERIALS

Efficacy Trial of a DNA/rAd5 HIV-1 Preventive Vaccine

(2013) Scott M. Hammer et al.   NEW ENGLAND JOURNAL OF MEDICINE

The Co-Delivery of Oxaliplatin Abrogates the Immunogenic Response to PEGylated siRNA-Lipoplex

(2013) Eman Alaaeldin et al.   PHARMACEUTICAL RESEARCH

Marker-free plasmids for biotechnological applications – implications and perspectives

(2013) Pedro H. Oliveira et al.   TRENDS IN BIOTECHNOLOGY

Absence of systemic toxicity changes following intramuscular administration of novel pSG2.HIVconsv DNA, ChAdV63.HIVconsv and MVA.HIVconsv vaccines to BALB/c mice

(2013) Beatrice Ondondo et al.   VACCINE

Nucleic acids and endosomal pattern recognition: how to tell friend from foe?

(2013) Eva Brencicova et al.   Frontiers in Cellular and Infection Microbiology

Pharmacological Characterization of a Novel ENaCα siRNA (GSK2225745) With Potential for the Treatment of Cystic Fibrosis

(2013) Kenneth L Clark et al.   Molecular Therapy-Nucleic Acids

AS1411 aptamer tagged PLGA-lecithin-PEG nanoparticles for tumor cell targeting and drug delivery

(2012) Athulya Aravind et al.   BIOTECHNOLOGY AND BIOENGINEERING

An open-label, phase 2 trial of RPI.4610 (angiozyme) in the treatment of metastatic breast cancer

(2012) Phuong Khanh Morrow et al.   CANCER

Toll-like receptor (TLR) 3 immune modulation by unformulated small interfering RNA or DNA and the role of CD14 (in TLR-mediated effects)

(2012) Cordula Weber et al.   IMMUNOLOGY

Inhibition of tissue factor pathway inhibitor by the aptamer BAX499 improves clotting of hemophilic blood and plasma

(2012) M. E. GORCZYCA et al.   JOURNAL OF THROMBOSIS AND HAEMOSTASIS

Assessing unintended hybridization-induced biological effects of oligonucleotides

(2012) Morten Lindow et al.   NATURE BIOTECHNOLOGY

In Vivo Sustained Release of siRNA from Solid Lipid Nanoparticles

(2011) Tatsiana Lobovkina et al.   ACS Nano

CpG DNA as a vaccine adjuvant

(2011) Christian Bode et al.   Expert Review of Vaccines

Anti-PEG IgM production by siRNA encapsulated in a PEGylated lipid nanocarrier is dependent on the sequence of the siRNA

(2011) Tatsuaki Tagami et al.   JOURNAL OF CONTROLLED RELEASE

Toxicity testing of the VEGF inhibitors bevacizumab, ranibizumab and pegaptanib in rats both with and without prior retinal ganglion cell damage

(2010) Sebastian Thaler et al.   ACTA OPHTHALMOLOGICA

Generation of human induced pluripotent stem cells by simple transient transfection of plasmid DNA encoding reprogramming factors

(2010) Karim Si-Tayeb et al.   BMC DEVELOPMENTAL BIOLOGY

In vitro immunotoxicity of ±2′-deoxy-3′-thiacytidine, a new and-HIV agent

(2010) G. LISIGNOLI et al.   CLINICAL AND EXPERIMENTAL IMMUNOLOGY

Therapeutic applications of ribozymes and riboswitches

(2010) Jérôme Mulhbacher et al.   CURRENT OPINION IN PHARMACOLOGY

Mechanistically Probing Lipid-siRNA Nanoparticle-associated Toxicities Identifies Jak Inhibitors Effective in Mitigating Multifaceted Toxic Responses

(2010) Weikang Tao et al.   MOLECULAR THERAPY

Aptamers as therapeutics

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

Immunotherapeutic applications of CpG oligodeoxynucleotide TLR9 agonists

(2009) Jörg Vollmer et al.   ADVANCED DRUG DELIVERY REVIEWS

Durable Protection from Herpes Simplex Virus-2 Transmission Following Intravaginal Application of siRNAs Targeting Both a Viral and Host Gene

(2009) Yichao Wu et al.   Cell Host & Microbe

Biodegradable calcium phosphate nanoparticle with lipid coating for systemic siRNA delivery

(2009) Jun Li et al.   JOURNAL OF CONTROLLED RELEASE

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

Drug Delivery–mediated Control of RNA Immunostimulation

(2009) David N. Nguyen et al.   MOLECULAR THERAPY

Evaluation of Efficacy, Biodistribution, and Inflammation for a Potent siRNA Nanoparticle: Effect of Dexamethasone Co-treatment

(2009) Marc T Abrams et al.   MOLECULAR THERAPY

First-in-human Mutation-targeted siRNA Phase Ib Trial of an Inherited Skin Disorder

(2009) Sancy A Leachman et al.   MOLECULAR THERAPY

Phase 2 gene therapy trial of an anti-HIV ribozyme in autologous CD34+ cells

(2009) Ronald T Mitsuyasu et al.   NATURE MEDICINE

Effective Treatment of Respiratory Alphaherpesvirus Infection Using RNA Interference

(2009) Amy Fulton et al.   PLoS One

Claudin-3 gene silencing with siRNA suppresses ovarian tumor growth and metastasis

(2009) Y.-H. Huang et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

TLR9 as a key receptor for the recognition of DNA☆

(2008) Y KUMAGAI et al.   ADVANCED DRUG DELIVERY REVIEWS

Targeting V600EB-Raf and Akt3 Using Nanoliposomal-Small Interfering RNA Inhibits Cutaneous Melanocytic Lesion Development

(2008) M. A. Tran et al.   CANCER RESEARCH

Nucleic Acids as Therapeutic Agents

(2008) Luis Alvarez-Salas   CURRENT TOPICS IN MEDICINAL CHEMISTRY

Folate-linked lipid-based nanoparticles for synthetic siRNA delivery in KB tumor xenografts

(2008) Takashi Yoshizawa et al.   EUROPEAN JOURNAL OF PHARMACEUTICS AND BIOPHARMACEUTICS

Intrathecal Raf-1-selective siRNA attenuates sustained morphine-mediated thermal hyperalgesia

(2008) Suneeta Tumati et al.   EUROPEAN JOURNAL OF PHARMACOLOGY

Custirsen (OGX-011): a second-generation antisense inhibitor of clusterin for the treatment of cancer

(2008) Kim N Chi et al.   EXPERT OPINION ON INVESTIGATIONAL DRUGS

An efficient and low immunostimulatory nanoparticle formulation for systemic siRNA delivery to the tumor

(2008) Sumio Chono et al.   JOURNAL OF CONTROLLED RELEASE

Eukaryotic Translation Initiation Factor 5A Small Interference RNA–Liposome Complexes Reduce Inflammation and Increase Survival in Murine Models of Severe Sepsis and Acute Lung Injury

(2008) Christopher C. Moore et al.   JOURNAL OF INFECTIOUS DISEASES

Novel role for the δ-opioid receptor in hypoxic preconditioning in rat retinas

(2008) Pai-Huei Peng et al.   JOURNAL OF NEUROCHEMISTRY

Coadministration of Cidofovir and Smallpox Vaccine Reduced Vaccination Side Effects but Interfered with Vaccine-Elicited Immune Responses and Immunity to Monkeypox

(2008) H. Wei et al.   JOURNAL OF VIROLOGY

Efficient Oncogene Silencing and Metastasis Inhibition via Systemic Delivery of siRNA

(2008) Shyh-Dar Li et al.   MOLECULAR THERAPY

Central Delivery of Dicer-substrate siRNA: A Direct Application for Pain Research

(2008) Louis Doré-Savard et al.   MOLECULAR THERAPY

Immunological Response to Peptide Nucleic Acid and its Peptide Conjugate Targeted to Transactivation Response (TAR) Region of HIV-1 RNA Genome

(2008) Alok Upadhyay et al.   OLIGONUCLEOTIDES

Structural requirements for uptake and recognition of CpG oligonucleotides

(2007) Klaus Heeg et al.   INTERNATIONAL JOURNAL OF MEDICAL MICROBIOLOGY

Tumor-targeted Delivery of siRNA by Self-assembled Nanoparticles

(2007) Shyh-Dar Li et al.   MOLECULAR THERAPY