4.5 Review

Lipid-based nucleic acid therapeutics with in vivo efficacy

相关参考文献

注意:仅列出部分参考文献,下载原文获取全部文献信息。
Review Pharmacology & Pharmacy

Oligonucleotide delivery to antigen presenting cells by using schizophyllan

Shinichi Mochizuki et al.

Summary: Schizophyllan (SPG) can form complexes with homo-polynucleotides and deliver functional oligonucleotides to antigen presenting cells through specific receptor Dectin-1, leading to therapeutic effects and immune responses.

DRUG METABOLISM AND PHARMACOKINETICS (2022)

Article Chemistry, Multidisciplinary

Targeting nucleic acid-based therapeutics to tumors: Challenges and strategies for polyplexes

Victoria C. Vetter et al.

Summary: This review summarizes the challenges and strategies for tumor-targeted nucleic acid delivery, with a focus on polyplexes as nanocarriers and their delivery options across multiple barriers into tumor cells.

JOURNAL OF CONTROLLED RELEASE (2022)

Review Chemistry, Medicinal

Biotechnological Evolution of siRNA Molecules: From Bench Tool to the Refined Drug

Danielle de Brito E. Cunha et al.

Summary: The depth and versatility of siRNA technologies make them suitable for disease targets that cannot be treated with small molecule drugs or require precise gene turn-off. However, the use of siRNA faces several challenges, which can be overcome by utilizing nanometric delivery carriers. siRNA has shown progress in clinical trials and has become a valuable asset for biopharmaceutical companies.

PHARMACEUTICALS (2022)

Article Chemistry, Multidisciplinary

Ultra-small lipid nanoparticles encapsulating sorafenib and midkine-siRNA selectively-eradicate sorafenib-resistant hepatocellular carcinoma in vivo

Mahmoud A. Younis et al.

Summary: The usLNPs, composed of a novel pH-sensitive lipid, phospholipids, and a targeting peptide, demonstrated enhanced tumor accumulation, selectivity, and in vivo gene silencing. This combination therapy synergistically eradicated HCC in mice at a low dose of SOR and showed promising potential for clinical applications.

JOURNAL OF CONTROLLED RELEASE (2021)

Article Biochemistry & Molecular Biology

Targeting alternative splicing by RNAi: from the differential impact on splice variants to triggering artificial pre-mRNA splicing

Armin Fuchs et al.

Summary: The study revealed the importance of RNA interference in regulating gene expression, while its interactions with alternative splicing were not well understood. By using amiRNA to knockdown various splice variants of target genes, it was found that some transcript isoforms escaped degradation due to their nuclear localization. The isoforms of alternatively spliced genes that are nuclear localized and sensitive to NMD mask the action of RNA interference.

NUCLEIC ACIDS RESEARCH (2021)

Article Biochemistry & Molecular Biology

The Performance of Minicircle DNA Versus Parental Plasmid in p53 Gene Delivery Into HPV-18-Infected Cervical Cancer Cells

Dalinda Eusebio et al.

Summary: The study demonstrated the superiority of mcDNA-p53 as a DNA vector in inhibiting the growth of HPV-18-infected cervical cancer cells, with higher p53 expression levels and transfection efficiency compared to traditional pDNA.

NUCLEIC ACID THERAPEUTICS (2021)

Article Chemistry, Multidisciplinary

The dawn of mRNA vaccines: The COVID-19 case

Rein Verbeke et al.

Summary: In less than a year since the COVID-19 pandemic outbreak, mRNA vaccines have made significant progress, with two receiving emergency use authorization and one moving to phase 3 clinical testing. These mRNA vaccines represent a new class of vaccine products, encoding the SARS-CoV-2 Spike glycoprotein to deliver mRNA to cells. The review provides a detailed overview of the composition, clinical performance, and design considerations of the most advanced mRNA vaccines, highlighting new insights and areas for future research and optimization.

JOURNAL OF CONTROLLED RELEASE (2021)

Review Engineering, Biomedical

Microfluidic formulation of nanoparticles for biomedical applications

Sarah J. Shepherd et al.

Summary: Nanomedicine has advanced significantly in clinical applications, with microfluidic devices showing potential for generating reproducible nanoparticle formulations. These formulations enhance biomedical applications with controlled physical properties, pointing towards future production scale-independent formulations through parallelization and multi-step reactions.

BIOMATERIALS (2021)

Article Chemistry, Multidisciplinary

Non-viral gene delivery of the oncotoxic protein NS1 for treatment of hepatocellular carcinoma

Dominik Witzigmann et al.

Summary: This study investigated a nonviral gene therapy approach targeting HCC with the NS1 protein, identifying PDK1 as a potential predictive biomarker for NS1-LPX gene therapy effectiveness. Experimental results demonstrated that the NS1-LPX therapeutic approach significantly reduced tumor growth and prolonged survival in an HCC xenograft mouse model.

JOURNAL OF CONTROLLED RELEASE (2021)

Review Pharmacology & Pharmacy

Non-Viral in Vitro Gene Delivery: It is Now Time to Set the Bar!

Nina Bono et al.

PHARMACEUTICS (2020)

Review Pharmacology & Pharmacy

Opportunities and Challenges in the Delivery of mRNA-Based Vaccines

Abishek Wadhwa et al.

PHARMACEUTICS (2020)

Review Medicine, Research & Experimental

Improved Nucleic Acid Therapy with Advanced Nanoscale Biotechnology

Yuhua Weng et al.

MOLECULAR THERAPY-NUCLEIC ACIDS (2020)

Review Chemistry, Multidisciplinary

Nanomedicines to Deliver mRNA: State of the Art and Future Perspectives

Itziar Gomez-Aguado et al.

NANOMATERIALS (2020)

Review Cell Biology

The powerful world of antisense oligonucleotides: From bench to bedside

Anais M. Quemener et al.

WILEY INTERDISCIPLINARY REVIEWS-RNA (2020)

Review Pharmacology & Pharmacy

Lipid nanoparticles for nucleic acid delivery: Current perspectives

Eleni Samaridou et al.

ADVANCED DRUG DELIVERY REVIEWS (2020)

Review Pharmacology & Pharmacy

Lipid nanoparticle technology for therapeutic gene regulation in the liver

Dominik Witzigmann et al.

ADVANCED DRUG DELIVERY REVIEWS (2020)

Article Biochemistry & Molecular Biology

A Thermostable mRNA Vaccine against COVID-19

Na-Na Zhang et al.

Article Multidisciplinary Sciences

An RNA vaccine drives immunity in checkpoint-inhibitor-treated melanoma

Ugur Sahin et al.

NATURE (2020)

Review Biotechnology & Applied Microbiology

Advances in oligonucleotide drug delivery

Thomas C. Roberts et al.

NATURE REVIEWS DRUG DISCOVERY (2020)

Article Medicine, General & Internal

An mRNA Vaccine against SARS-CoV-2-Preliminary Report

L. A. Jackson et al.

NEW ENGLAND JOURNAL OF MEDICINE (2020)

Review Biochemistry & Molecular Biology

Therapeutic siRNA: state of the art

Bo Hu et al.

SIGNAL TRANSDUCTION AND TARGETED THERAPY (2020)

Review Pharmacology & Pharmacy

Strategies, design, and chemistry in siRNA delivery systems

Yizhou Dong et al.

ADVANCED DRUG DELIVERY REVIEWS (2019)

Article Biotechnology & Applied Microbiology

Chemical modification of PS-ASO therapeutics reduces cellular protein-binding and improves the therapeutic index

Wen Shen et al.

NATURE BIOTECHNOLOGY (2019)

Review Respiratory System

Emerging gene therapies for cystic fibrosis

Kamran M. Miah et al.

EXPERT REVIEW OF RESPIRATORY MEDICINE (2019)

Review Chemistry, Multidisciplinary

Lipid Nanoparticle Technology for Clinical Translation of siRNA Therapeutics

Jayesh A. Kulkarni et al.

ACCOUNTS OF CHEMICAL RESEARCH (2019)

Letter Biochemical Research Methods

Caution: Plasmid DNA topology affects luciferase assay reproducibility and outcomes

Emma Tudini et al.

BIOTECHNIQUES (2019)

Review Biochemistry & Molecular Biology

Peptide nucleic acid (PNA) and its applications in chemical biology, diagnostics, and therapeutics

Jacques Saarbach et al.

CURRENT OPINION IN CHEMICAL BIOLOGY (2019)

Review Biochemistry & Molecular Biology

mRNA as novel technology for passive immunotherapy

Thomas Schlake et al.

CELLULAR AND MOLECULAR LIFE SCIENCES (2019)

Review Biotechnology & Applied Microbiology

Delivery technologies for cancer immunotherapy

Rachel S. Riley et al.

NATURE REVIEWS DRUG DISCOVERY (2019)

Article Chemistry, Medicinal

ARB-1740, a RNA Interference Therapeutic for Chronic Hepatitis B Infection

Emily P. Thi et al.

ACS INFECTIOUS DISEASES (2019)

Review Chemistry, Multidisciplinary

Lipid-Based DNA Therapeutics: Hallmarks of Non-Viral Gene Delivery

Jonas Buck et al.

ACS NANO (2019)

Review Pharmacology & Pharmacy

Developing small activating RNA as a therapeutic: current challenges and promises

Albert Kwok et al.

THERAPEUTIC DELIVERY (2019)

Review Genetics & Heredity

miR-7 Replacement Therapy in Parkinson's Disease

Ricardo Titze-de-Almeida et al.

CURRENT GENE THERAPY (2018)

Review Pharmacology & Pharmacy

Locked nucleic acid: modality, diversity, and drug discovery

Peter H. Hagedorn et al.

DRUG DISCOVERY TODAY (2018)

Article Biotechnology & Applied Microbiology

Lipid Nanoparticle-Delivered Chemically Modified mRNA Restores Chloride Secretion in Cystic Fibrosis

Ema Robinson et al.

MOLECULAR THERAPY (2018)

Article Biotechnology & Applied Microbiology

Self-Amplifying RNA Vaccines Give Equivalent Protection against Influenza to mRNA Vaccines but at Much Lower Doses

Annette B. Vogel et al.

MOLECULAR THERAPY (2018)

Article Biotechnology & Applied Microbiology

Targeted mRNA Therapy for Ornithine Transcarbamylase Deficiency

Mary G. Prieve et al.

MOLECULAR THERAPY (2018)

Review Biotechnology & Applied Microbiology

mRNA vaccines - a new era in vaccinology

Norbert Pardi et al.

NATURE REVIEWS DRUG DISCOVERY (2018)

Article Medicine, General & Internal

Patisiran, an RNAi Therapeutic, for Hereditary Transthyretin Amyloidosis

D. Adams et al.

NEW ENGLAND JOURNAL OF MEDICINE (2018)

Review Biochemistry & Molecular Biology

Lipid Nanoparticles Enabling Gene Therapies: From Concepts to Clinical Utility

Jayesh A. Kulkarni et al.

NUCLEIC ACID THERAPEUTICS (2018)

Review Microbiology

Antisense Phosphorodiamidate Morpholino Oligomers as Novel Antiviral Compounds

Yuchen Nan et al.

FRONTIERS IN MICROBIOLOGY (2018)

Review Chemistry, Multidisciplinary

Synthetic materials at the forefront of gene delivery

Irene Lostale-Seijo et al.

NATURE REVIEWS CHEMISTRY (2018)

Review Biochemistry & Molecular Biology

Development of MTL-CEBPA: Small Activating RNA Drug for Hepatocellular Carcinoma

Ryan L. Setten et al.

CURRENT PHARMACEUTICAL BIOTECHNOLOGY (2018)

Review Chemistry, Multidisciplinary

mRNA therapeutics deliver a hopeful message

Zifu Zhong et al.

NANO TODAY (2018)

Review Biotechnology & Applied Microbiology

Cornerstones of CRISPR-Cas in drug discovery and therapy

Christof Fellmann et al.

NATURE REVIEWS DRUG DISCOVERY (2017)

Review Biotechnology & Applied Microbiology

Future of rAAV Gene Therapy: Platform for RNAi, Gene Editing, and Beyond

Paul N. Valdmanis et al.

HUMAN GENE THERAPY (2017)

Article Biotechnology & Applied Microbiology

Lipid Nanoparticle Systems for Enabling Gene Therapies

Pieter R. Cullis et al.

MOLECULAR THERAPY (2017)

Article Nanoscience & Nanotechnology

Design of lipid nanoparticles for in vitro and in vivo delivery of plasmid DNA

Jayesh A. Kulkarni et al.

NANOMEDICINE-NANOTECHNOLOGY BIOLOGY AND MEDICINE (2017)

Article Multidisciplinary Sciences

Exosomes facilitate therapeutic targeting of oncogenic KRAS in pancreatic cancer

Sushrut Kamerkar et al.

NATURE (2017)

Article Multidisciplinary Sciences

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

Norbert Pardi et al.

NATURE (2017)

Article Biotechnology & Applied Microbiology

Structure-guided chemical modification of guide RNA enables potent non-viral in vivo genome editing

Hao Yin et al.

NATURE BIOTECHNOLOGY (2017)

Article Multidisciplinary Sciences

Barcoded nanoparticles for high throughput in vivo discovery of targeted therapeutics

James E. Dahlman et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2017)

Review Nanoscience & Nanotechnology

Tools for translation: non-viral materials for therapeutic mRNA delivery

Khalid A. Hajj et al.

NATURE REVIEWS MATERIALS (2017)

Review Cell Biology

Strategies to Inhibit Myc and Their Clinical Applicability

Jonathan R. Whitfield et al.

FRONTIERS IN CELL AND DEVELOPMENTAL BIOLOGY (2017)

Article Biochemistry & Molecular Biology

Modified mRNA Vaccines Protect against Zika Virus Infection

Justin M. Richner et al.

Review Pharmacology & Pharmacy

The role of helper lipids in lipid nanoparticles (LNPs) designed for oligonucleotide delivery

Xinwei Cheng et al.

ADVANCED DRUG DELIVERY REVIEWS (2016)

Review Pharmacology & Pharmacy

Recent progress in development of siRNA delivery vehicles for cancer therapy

Hyun Jin Kim et al.

ADVANCED DRUG DELIVERY REVIEWS (2016)

Article Genetics & Heredity

Challenges and Successes Using Nanomedicines for Aerosol Delivery to the Airways

P. Resnier et al.

CURRENT GENE THERAPY (2016)

Review Biotechnology & Applied Microbiology

Intralesional immunotherapy as a strategy to treat melanoma

Noura Nouri et al.

EXPERT OPINION ON BIOLOGICAL THERAPY (2016)

Article Chemistry, Multidisciplinary

RGD liposome-protamine-siRNA (LPR) nanoparticles targeting PAX3-FOXO1 for alveolar rhabdomyosarcoma therapy

Venkatesh Rengaswamy et al.

JOURNAL OF CONTROLLED RELEASE (2016)

Article Chemistry, Multidisciplinary

Influence of particle size on the in vivo potency of lipid nanoparticle formulations of siRNA

Sam Chen et al.

JOURNAL OF CONTROLLED RELEASE (2016)

Article Gastroenterology & Hepatology

Novel pH-sensitive multifunctional envelope-type nanodevice for siRNA-based treatments for chronic HBV infection

Naoki Yamamoto et al.

JOURNAL OF HEPATOLOGY (2016)

Review Chemistry, Multidisciplinary

Emerging Frontiers in Drug Delivery

Mark W. Tibbitt et al.

JOURNAL OF THE AMERICAN CHEMICAL SOCIETY (2016)

Article Biotechnology & Applied Microbiology

Preclinical Justification of pbi-shRNA EWS/FLI1 Lipoplex (LPX) Treatment for Ewing's Sarcoma

Donald D. Rao et al.

MOLECULAR THERAPY (2016)

Article Biotechnology & Applied Microbiology

A Phase I Study of a Tumor-targeted Systemic Nanodelivery System, SGT-94, in Genitourinary Cancers

Arlene Siefker-Radtke et al.

MOLECULAR THERAPY (2016)

Editorial Material Biotechnology & Applied Microbiology

siRNA Therapeutics for Primary Hyperoxaluria: A Beginning

Dawn S. Milliner

MOLECULAR THERAPY (2016)

Article Biotechnology & Applied Microbiology

Glycolate Oxidase Is a Safe and Efficient Target for Substrate Reduction Therapy in a Mouse Model of Primary Hyperoxaluria Type I

Cristina Martin-Higueras et al.

MOLECULAR THERAPY (2016)

Review Biology

CRISPR-Cas: biology, mechanisms and relevance

Frank Hille et al.

PHILOSOPHICAL TRANSACTIONS OF THE ROYAL SOCIETY B-BIOLOGICAL SCIENCES (2016)

Article Medicine, General & Internal

Experimental Treatment of Ebola Virus Disease with TKM-130803: A Single-Arm Phase 2 Clinical Trial

Jake Dunning et al.

PLOS MEDICINE (2016)

Article Multidisciplinary Sciences

Liposome production by microfluidics: potential and limiting factors

Dario Carugo et al.

SCIENTIFIC REPORTS (2016)

Meeting Abstract Oncology

A phase I/II study of TKM-080301, a PLK1-targeted RNAi in patients with adrenocortical cancer (ACC).

Michael J. Demeure et al.

JOURNAL OF CLINICAL ONCOLOGY (2016)

Review Pharmacology & Pharmacy

Delivery of oligonucleotides with lipid nanoparticles

Yuhua Wang et al.

ADVANCED DRUG DELIVERY REVIEWS (2015)

Article Computer Science, Interdisciplinary Applications

Flow regimes in T-shaped micro-mixers

Tommaso Andreussi et al.

COMPUTERS & CHEMICAL ENGINEERING (2015)

Article Chemistry, Medicinal

Synthetic Nucleic Acid Delivery Systems: Present and Perspectives

Bogdan Draghici et al.

JOURNAL OF MEDICINAL CHEMISTRY (2015)

Article Chemistry, Physical

Microfluidic Mixing: A General Method for Encapsulating Macromolecules in Lipid Nanoparticle Systems

Alex K. K. Leung et al.

JOURNAL OF PHYSICAL CHEMISTRY B (2015)

Article Biotechnology & Applied Microbiology

Phase 1 Trial of Bi-shRNA STMN1 BIV in Refractory Cancer

Minal Barve et al.

MOLECULAR THERAPY (2015)

Article Chemistry, Multidisciplinary

An Orthogonal Array Optimization of Lipid-like Nanoparticles for mRNA Delivery in Vivo

Bin Li et al.

NANO LETTERS (2015)

Article Nanoscience & Nanotechnology

A tumor-targeting p53 nanodelivery system limits chemoresistance to temozolomide prolonging survival in a mouse model of glioblastoma multiforme

Sang-Soo Kim et al.

NANOMEDICINE-NANOTECHNOLOGY BIOLOGY AND MEDICINE (2015)

Article Multidisciplinary Sciences

Lipid nanoparticle siRNA treatment of Ebola-virus-Makona-infected nonhuman primates

Emily P. Thi et al.

NATURE (2015)

Article Genetics & Heredity

Efficacy and safety of patisiran for familial amyloidotic polyneuropathy: a phase II multi-dose study

Ole B. Suhr et al.

ORPHANET JOURNAL OF RARE DISEASES (2015)

Review Medicine, Research & Experimental

siRNA Versus miRNA as Therapeutics for Gene Silencing

Jenny K. W. Lam et al.

MOLECULAR THERAPY-NUCLEIC ACIDS (2015)

Article Pharmacology & Pharmacy

Discontinued in 2013: oncology drugs

Robert Williams

EXPERT OPINION ON INVESTIGATIONAL DRUGS (2015)

Review Pharmacology & Pharmacy

Gene therapy and DNA delivery systems

D. Ibraheem et al.

INTERNATIONAL JOURNAL OF PHARMACEUTICS (2014)

Review Genetics & Heredity

Non-viral vectors for gene-based therapy

Hao Yin et al.

NATURE REVIEWS GENETICS (2014)

Article Multidisciplinary Sciences

Lipopeptide nanoparticles for potent and selective siRNA delivery in rodents and nonhuman primates

Yizhou Dong et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2014)

Review Multidisciplinary Sciences

The new frontier of genome engineering with CRISPR-Cas9

Jennifer A. Doudna et al.

SCIENCE (2014)

Review Biochemistry & Molecular Biology

A critical analysis of codon optimization in human therapeutics

Vincent P. Mauro et al.

TRENDS IN MOLECULAR MEDICINE (2014)

Review Pharmacology & Pharmacy

Liposomal drug delivery systems: From concept to clinical applications

Theresa M. Allen et al.

ADVANCED DRUG DELIVERY REVIEWS (2013)

Article Biotechnology & Applied Microbiology

Phase I Study of a Systemically Delivered p53 Nanoparticle in Advanced Solid Tumors

Neil Senzer et al.

MOLECULAR THERAPY (2013)

Article Biotechnology & Applied Microbiology

Biodegradable Lipids Enabling Rapidly Eliminated Lipid Nanoparticles for Systemic Delivery of RNAi Therapeutics

Martin A. Maier et al.

MOLECULAR THERAPY (2013)

Article Biotechnology & Applied Microbiology

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

Jerome Gilleron et al.

NATURE BIOTECHNOLOGY (2013)

Article Medicine, General & Internal

Safety and Efficacy of RNAi Therapy for Transthyretin Amyloidosis

Teresa Coelho et al.

NEW ENGLAND JOURNAL OF MEDICINE (2013)

Review Biochemistry & Molecular Biology

Complexity of the Alternative Splicing Landscape in Plants

Anireddy S. N. Reddy et al.

PLANT CELL (2013)

Article Multidisciplinary Sciences

Multiparametric approach for the evaluation of lipid nanoparticles for siRNA delivery

Christopher A. Alabi et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2013)

Article Multidisciplinary Sciences

Multiplex Genome Engineering Using CRISPR/Cas Systems

Le Cong et al.

SCIENCE (2013)

Article Medicine, Research & Experimental

Influence of Polyethylene Glycol Lipid Desorption Rates on Pharmacokinetics and Pharmacodynamics of siRNA Lipid Nanoparticles

Barbara L. Mui et al.

MOLECULAR THERAPY-NUCLEIC ACIDS (2013)

Article Pharmacology & Pharmacy

Cholesterol domains enhance transfection

Jamie L. Betker et al.

THERAPEUTIC DELIVERY (2013)

Review Chemistry, Multidisciplinary

Recent Advances in Nonviral Vectors for Gene Delivery

Xia Guo et al.

ACCOUNTS OF CHEMICAL RESEARCH (2012)

Review Chemistry, Multidisciplinary

Synthetic Gene Transfer Vectors II: Back to the Future

Jean-Paul Behr

ACCOUNTS OF CHEMICAL RESEARCH (2012)

Article Chemistry, Multidisciplinary

Maximizing the Potency of siRNA Lipid Nanoparticles for Hepatic Gene Silencing In Vivo

Muthusamy Jayaraman et al.

ANGEWANDTE CHEMIE-INTERNATIONAL EDITION (2012)

Article Biochemistry & Molecular Biology

Transfection efficiency boost of cholesterol-containing lipoplexes

Daniela Pozzi et al.

BIOCHIMICA ET BIOPHYSICA ACTA-BIOMEMBRANES (2012)

Review Biochemistry & Molecular Biology

Lipophosphonate/lipophosphoramidates: A family of synthetic vectors efficient for gene delivery

Mathieu Berchel et al.

BIOCHIMIE (2012)

Article Engineering, Biomedical

The gene transfection properties of a lipophosphoramidate derivative with two phytanyl chains

Mattias F. Lindberg et al.

BIOMATERIALS (2012)

Review Chemistry, Multidisciplinary

Liposome based systems for systemic siRNA delivery: Stability in blood sets the requirements for optimal carrier design

Kevin Buyens et al.

JOURNAL OF CONTROLLED RELEASE (2012)

Article Chemistry, Physical

Lipid Nanoparticles Containing siRNA Synthesized by Microfluidic Mixing Exhibit an Electron-Dense Nanostructured Core

Alex K. K. Leung et al.

JOURNAL OF PHYSICAL CHEMISTRY C (2012)

Article Chemistry, Multidisciplinary

Rapid Discovery of Potent siRNA-Containing Lipid Nanoparticles Enabled by Controlled Microfluidic Formulation

Delai Chen et al.

JOURNAL OF THE AMERICAN CHEMICAL SOCIETY (2012)

Article Biotechnology & Applied Microbiology

UU/UA Dinucleotide Frequency Reduction in Coding Regions Results in Increased mRNA Stability and Protein Expression

Maher Al-Saif et al.

MOLECULAR THERAPY (2012)

Review Biochemistry & Molecular Biology

Translational control by changes in poly(A) tail length: recycling mRNAs

Laure Weill et al.

NATURE STRUCTURAL & MOLECULAR BIOLOGY (2012)

Article Multidisciplinary Sciences

A Programmable Dual-RNA-Guided DNA Endonuclease in Adaptive Bacterial Immunity

Martin Jinek et al.

SCIENCE (2012)

Article Medicine, Research & Experimental

Microfluidic Synthesis of Highly Potent Limit-size Lipid Nanoparticles for In Vivo Delivery of siRNA

Nathan M. Belliveau et al.

MOLECULAR THERAPY-NUCLEIC ACIDS (2012)

Article Biochemistry & Molecular Biology

In vivo Safety and Antitumor Efficacy of Bifunctional Small Hairpin RNAs Specific for the Human Stathmin 1 Oncoprotein

Anagha P. Phadke et al.

DNA AND CELL BIOLOGY (2011)

Article Biotechnology & Applied Microbiology

The impact of DNA topology on polyplex uptake and transfection efficiency in mammalian cells

Arjun Dhanoya et al.

JOURNAL OF BIOTECHNOLOGY (2011)

Article Biochemistry & Molecular Biology

Improved specificity of gene silencing by siRNAs containing unlocked nucleobase analogs

Narendra Vaish et al.

NUCLEIC ACIDS RESEARCH (2011)

Article Biochemistry & Molecular Biology

Targeted hepatocellular carcinoma proapoptotic BikDD gene therapy

L-Y Li et al.

ONCOGENE (2011)

Article Chemistry, Multidisciplinary

Microfluidic Mixing and the Formation of Nanoscale Lipid Vesicles

Andreas Jahn et al.

ACS NANO (2010)

Review Pharmacology & Pharmacy

RNA Targeting Therapeutics: Molecular Mechanisms of Antisense Oligonucleotides as a Therapeutic Platform

C. Frank Bennett et al.

ANNUAL REVIEW OF PHARMACOLOGY AND TOXICOLOGY (2010)

Review Biochemistry & Molecular Biology

Cationic lipid-mediated nucleic acid delivery: beyond being cationic

N. Madhusudhana Rao

CHEMISTRY AND PHYSICS OF LIPIDS (2010)

Article Biochemistry & Molecular Biology

MicroRNA: Biogenesis, Function and Role in Cancer

Leigh-Ann MacFarlane et al.

CURRENT GENOMICS (2010)

Article Pharmacology & Pharmacy

DC-Chol/DOPE cationic liposomes: A comparative study of the influence factors on plasmid pDNA and siRNA gene delivery

Yang Zhang et al.

INTERNATIONAL JOURNAL OF PHARMACEUTICS (2010)

Article Biochemistry & Molecular Biology

Three-dimensional structured illumination microscopy of liver sinusoidal endothelial cell fenestrations

Victoria C. Cogger et al.

JOURNAL OF STRUCTURAL BIOLOGY (2010)

Article Biochemistry & Molecular Biology

Utilization of unlocked nucleic acid (UNA) to enhance siRNA performance in vitro and in vivo

Maria B. Laursen et al.

MOLECULAR BIOSYSTEMS (2010)

Article Biotechnology & Applied Microbiology

Rational design of cationic lipids for siRNA delivery

Sean C. Semple et al.

NATURE BIOTECHNOLOGY (2010)

Article Multidisciplinary Sciences

Lipid-like materials for low-dose, in vivo gene silencing

Kevin T. Love et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2010)

Article Immunology

Sequence determinants of innate immune activation by short interfering RNAs

Amber Goodchild et al.

BMC IMMUNOLOGY (2009)

Review Pharmacology & Pharmacy

Physical methods of nucleic acid transfer: general concepts and applications

Julien Villemejane et al.

BRITISH JOURNAL OF PHARMACOLOGY (2009)

Article Biotechnology & Applied Microbiology

Early RB94-produced cytotoxicity in cancer cells is independent of caspase activation or 50 kb DNA fragmentation

J. Zhou et al.

CANCER GENE THERAPY (2009)

Article Biochemistry & Molecular Biology

Importin 8 Is a Gene Silencing Factor that Targets Argonaute Proteins to Distinct mRNAs

Lasse Weinmann et al.

Review Biochemistry & Molecular Biology

Origins and Mechanisms of miRNAs and siRNAs

Richard W. Carthew et al.

Review Chemistry, Multidisciplinary

Advances in gene delivery through molecular design of cationic lipids

Santanu Bhattacharya et al.

CHEMICAL COMMUNICATIONS (2009)

Review Chemistry, Multidisciplinary

Nonviral Vectors for Gene Delivery

Meredith A. Mintzer et al.

CHEMICAL REVIEWS (2009)

Review Biochemistry & Molecular Biology

Progress and Prospects: The design and production of plasmid vectors

D. R. Gill et al.

GENE THERAPY (2009)

Review Cell Biology

Many roads to maturity: microRNA biogenesis pathways and their regulation

Julia Winter et al.

NATURE CELL BIOLOGY (2009)

Review Biotechnology & Applied Microbiology

Knocking down barriers: advances in siRNA delivery

Kathryn A. Whitehead et al.

NATURE REVIEWS DRUG DISCOVERY (2009)

Review Pharmacology & Pharmacy

Conventional and Dense Gas Techniques for the Production of Liposomes: A Review

Louise A. Meure et al.

AAPS PHARMSCITECH (2008)

Article Biochemistry & Molecular Biology

Synthesis of 2 '-O-modified adenosine building blocks and application for RNA interference

Dalibor Odadzic et al.

BIOORGANIC & MEDICINAL CHEMISTRY (2008)

Article Medicine, Research & Experimental

Pharmacokinetics and biodistribution of nanoparticles

Shyh-Dar Li et al.

MOLECULAR PHARMACEUTICS (2008)

Article Biotechnology & Applied Microbiology

CpG-free plasmids confer reduced inflammation and sustained pulmonary gene expression

Stephen C. Hyde et al.

NATURE BIOTECHNOLOGY (2008)

Article Biotechnology & Applied Microbiology

Resolution of liver cirrhosis using vitamin A-coupled liposomes to deliver siRNA against a collagen-specific chaperone

Yasushi Sato et al.

NATURE BIOTECHNOLOGY (2008)

Article Biotechnology & Applied Microbiology

A combinatorial library of lipid-like materials for delivery of RNAi therapeutics

Akin Akinc et al.

NATURE BIOTECHNOLOGY (2008)

Article Multidisciplinary Sciences

Therapeutic RNAi targeting PCSK9 acutely lowers plasma cholesterol in rodents and LDL cholesterol in nonhuman primates

Maria Frank-Kamenetsky et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2008)

Article Biotechnology & Applied Microbiology

Renal clearance of quantum dots

Hak Soo Choi et al.

NATURE BIOTECHNOLOGY (2007)

Article Biotechnology & Applied Microbiology

Systemic and specific delivery of small interfering RNAs to the liver mediated by apolipoprotein A-I

Soo In Kim et al.

MOLECULAR THERAPY (2007)

Article Biochemical Research Methods

A protocol for designing siRNAs with high functionality and specificity

Amanda Birmingham et al.

NATURE PROTOCOLS (2007)

Review Oncology

Nonviral delivery vehicles for use in short hairpin RNA-based cancer therapies

John S. Vorhies et al.

EXPERT REVIEW OF ANTICANCER THERAPY (2007)

Review Chemistry, Multidisciplinary

Lipid-based nanoparticles for nucleic acid delivery

Weijun Li et al.

PHARMACEUTICAL RESEARCH (2007)

Article Oncology

Phase 2 trial of Allovectin-7 in advanced metastatic melanoma

Rene Gonzalez et al.

MELANOMA RESEARCH (2006)

Article Immunology

Nucleic acid agonists for toll-like receptor 7 are defined by the presence of uridine ribonucleotides

Sandra S. Diebold et al.

EUROPEAN JOURNAL OF IMMUNOLOGY (2006)

Review Pharmacology & Pharmacy

Vector-based RNAi approaches for stable, inducible and genome-wide screens

Gwen D. Fewell et al.

DRUG DISCOVERY TODAY (2006)

Article Biochemistry & Molecular Biology

A novel siRNA-lipoplex technology for RNA interference in the mouse vascular endothelium

A. Santel et al.

GENE THERAPY (2006)

Article Chemistry, Multidisciplinary

Development and in vitro validation of a targeted delivery vehicle for DNA vaccines

Silke S. Talsma et al.

JOURNAL OF CONTROLLED RELEASE (2006)

Article Multidisciplinary Sciences

RNAi-mediated gene silencing in non-human primates

TS Zimmermann et al.

NATURE (2006)

Article Pharmacology & Pharmacy

Antisense oligonucleotides: From design to therapeutic application

JHP Chan et al.

CLINICAL AND EXPERIMENTAL PHARMACOLOGY AND PHYSIOLOGY (2006)

Article Biotechnology & Applied Microbiology

Use of lipoplex-induced nuclear factor-kappa B activation to enhance transgene expression by lipoplex in mouse tung

T Kuramoto et al.

JOURNAL OF GENE MEDICINE (2006)

Review Chemistry, Multidisciplinary

Synthetic, self-assembly ABCD nanoparticles; a structural paradigm for viable synthetic non-viral vectors

K Kostarelos et al.

CHEMICAL SOCIETY REVIEWS (2005)

Article Chemistry, Multidisciplinary

Cationic lipid saturation influences intracellular delivery of encapsulated nucleic acids

J Heyes et al.

JOURNAL OF CONTROLLED RELEASE (2005)

Article Biotechnology & Applied Microbiology

Potent and persistent in vivo anti-HBV activity of chemically modified siRNAs

DV Morrissey et al.

NATURE BIOTECHNOLOGY (2005)

Article Biochemistry & Molecular Biology

The role of apolipoprotein E in the elimination of liposomes from blood by hepatocytes in the mouse

XD Yan et al.

BIOCHEMICAL AND BIOPHYSICAL RESEARCH COMMUNICATIONS (2005)

Article Pharmacology & Pharmacy

Immunogenicity and rapid blood clearance of liposomes containing polyethylene glycol-lipid conjugates and nucleic acid

SC Semple et al.

JOURNAL OF PHARMACOLOGY AND EXPERIMENTAL THERAPEUTICS (2005)

Review Pharmacology & Pharmacy

DNA-based therapeutics and DNA delivery systems: A comprehensive review

SD Patil et al.

AAPS JOURNAL (2005)

Article Biochemistry & Molecular Biology

Determination of the role of the human RNase H1 in the pharmacology of DNA-like antisense drugs

HJ Wu et al.

JOURNAL OF BIOLOGICAL CHEMISTRY (2004)

Review Biotechnology & Applied Microbiology

DNA double-strand break repair by homologous recombination

A Dudás et al.

MUTATION RESEARCH-REVIEWS IN MUTATION RESEARCH (2004)

Article Biochemistry & Molecular Biology

PKN3 is required for malignant prostate cell growth downstream of activated PI 3-kinase

F Leenders et al.

EMBO JOURNAL (2004)

Article Chemistry, Medicinal

Pyridinium cationic lipids in gene delivery: A structure-activity correlation study

MA Ilies et al.

JOURNAL OF MEDICINAL CHEMISTRY (2004)

Article Biochemistry & Molecular Biology

DOTAP/DOPE and DC-Chol/DOPE lipoplexes for gene delivery: zeta potential measurements and electron spin resonance spectra

L Ciani et al.

BIOCHIMICA ET BIOPHYSICA ACTA-BIOMEMBRANES (2004)

Article Biochemistry & Molecular Biology

RNA interference using boranophosphate siRNAs: structure-activity relationships

AHS Hall et al.

NUCLEIC ACIDS RESEARCH (2004)

Article Biotechnology & Applied Microbiology

Tissue distribution of liposome-mediated epidermal growth factor receptor antisense gene therapy

SM Thomas et al.

CANCER GENE THERAPY (2003)

Review Biochemistry & Molecular Biology

Antisense technologies - Improvement through novel chemical modifications

J Kurreck

EUROPEAN JOURNAL OF BIOCHEMISTRY (2003)

Review Chemistry, Multidisciplinary

Gemini surfactants: New synthetic vectors for gene transfection

AJ Kirby et al.

ANGEWANDTE CHEMIE-INTERNATIONAL EDITION (2003)

Review Biochemical Research Methods

Non-viral gene delivery systems

ME Davis

CURRENT OPINION IN BIOTECHNOLOGY (2002)

Article Multidisciplinary Sciences

Chaotic mixer for microchannels

AD Stroock et al.

SCIENCE (2002)

Review Pharmacology & Pharmacy

Cationic lipids in gene delivery: Principles, vector design and therapeutical applications

MA Ilies et al.

CURRENT PHARMACEUTICAL DESIGN (2002)

Article Biochemistry & Molecular Biology

Systemic p53 gene therapy of cancer with immunolipoplexes targeted by anti-transferrin receptor scFv

L Xu et al.

MOLECULAR MEDICINE (2001)

Article Biochemistry & Molecular Biology

On the mechanism whereby cationic lipids promote intracellular delivery of polynucleic acids

IM Hafez et al.

GENE THERAPY (2001)

Article Multidisciplinary Sciences

Duplexes of 21-nucleotide RNAs mediate RNA interference in cultured mammalian cells

SM Elbashir et al.

NATURE (2001)

Review Biotechnology & Applied Microbiology

Nonviral vectors in the new millennium: Delivery barriers in gene transfer

M Nishikawa et al.

HUMAN GENE THERAPY (2001)

Article Biotechnology & Applied Microbiology

A clinical inflammatory syndrome attributable to aerosolized lipid-DNA administration in cystic fibrosis

FE Ruiz et al.

HUMAN GENE THERAPY (2001)

Article Biochemistry & Molecular Biology

Repeat administration of DNA/liposomes to the nasal epithelium of patients with cystic fibrosis

SC Hyde et al.

GENE THERAPY (2000)

Article Multidisciplinary Sciences

Potent and nontoxic antisense oligonucleotides containing locked nucleic acids

C Wahlestedt et al.

PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA (2000)

Article Pharmacology & Pharmacy

Optimization of cationic lipid/DNA complexes for systemic gene transfer to tumor lesions

K Anwer et al.

JOURNAL OF DRUG TARGETING (2000)