At the Intersection of Biomaterials and Gene Therapy: Progress in Non-viral Delivery of Nucleic Acids

Impact of polymer-TLR-7/8 agonist (adjuvant) morphology on the potency and mechanism of CD8 T cell induction

(2019) Geoffrey M. Lynn et al.   BIOMACROMOLECULES

Self-assembled peptide–poloxamine nanoparticles enable in vitro and in vivo genome restoration for cystic fibrosis

(2019) Shan Guan et al.   Nature Nanotechnology

Modularly engineered injectable hybrid hydrogels based on protein-polymer network as potent immunologic adjuvant in vivo

(2019) V.H. Giang Phan et al.   BIOMATERIALS

Designing immunostimulatory double stranded messenger RNA with maintained translational activity through hybridization with poly A sequences for effective vaccination

(2018) Satoshi Uchida et al.   BIOMATERIALS

Non-viral RNA chimeric antigen receptor modified T cells in patients with Hodgkin lymphoma

(2018) Jakub Svoboda et al.   BLOOD

Delivering CRISPR: a review of the challenges and approaches

(2018) Christopher A. Lino et al.   DRUG DELIVERY

Incorporation of Synthetic mRNA in Injectable Chitosan-Alginate Hybrid Hydrogels for Local and Sustained Expression of Exogenous Proteins in Cells

(2018) Heidrun Steinle et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Cationic polymers for non-viral gene delivery to human T cells

(2018) Brynn R. Olden et al.   JOURNAL OF CONTROLLED RELEASE

Cutting Edge: Piezo1 Mechanosensors Optimize Human T Cell Activation

(2018) Chinky Shiu Chen Liu et al.   JOURNAL OF IMMUNOLOGY

Efficient Non-viral Gene Delivery into Human Hematopoietic Stem Cells by Minicircle Sleeping Beauty Transposon Vectors

(2018) Marta Holstein et al.   MOLECULAR THERAPY

Scaffolds that mimic antigen-presenting cells enable ex vivo expansion of primary T cells

(2018) Alexander S Cheung et al.   NATURE BIOTECHNOLOGY

Determinants of response and resistance to CD19 chimeric antigen receptor (CAR) T cell therapy of chronic lymphocytic leukemia

(2018) Joseph A. Fraietta et al.   NATURE MEDICINE

A modular platform for targeted RNAi therapeutics

(2018) Ranit Kedmi et al.   Nature Nanotechnology

Molecular spherical nucleic acids

(2018) Hui Li et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Nonviral gene editing via CRISPR/Cas9 delivery by membrane-disruptive and endosomolytic helical polypeptide

(2018) Hong-Xia Wang et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

An improved, chemically modified RNA encoding BMP-2 enhances osteogenesis in vitro and in vivo

(2018) Wen Zhang et al.   TISSUE ENGINEERING PART A

DNA Nanostructure-Based Systems for Intelligent Delivery of Therapeutic Oligonucleotides

(2018) Qinqin Hu et al.   Advanced Healthcare Materials

Therapeutic applications of group I intron-based trans -splicing ribozymes

(2018) Chang Ho Lee et al.   Wiley Interdisciplinary Reviews-RNA

Self-Crosslinking Lipopeptide/DNA/PEGylated Particles: A New Platform for DNA Vaccination Designed for Assembly in Aqueous Solution

(2018) Joan K. Ho et al.   Molecular Therapy-Nucleic Acids

Additive polyplexes to undertake siRNA therapy against CDC20 and survivin in breast cancer cells

(2018) Manoj B Parmar et al.   BIOMACROMOLECULES

Plasmid DNA Size Does Affect Nonviral Gene Delivery Efficiency in Stem Cells

(2018) Sofia Ribeiro et al.   Cellular Reprogramming

In vivo generation of human CD19‐CAR T cells results in B‐cell depletion and signs of cytokine release syndrome

(2018) Anett Pfeiffer et al.   EMBO Molecular Medicine

Scaffold-mediated delivery for non-viral mRNA vaccines

(2018) Ruying Chen et al.   GENE THERAPY

Immunomodulating Nanomedicine for Cancer Therapy

(2018) Zhigang Liu et al.   NANO LETTERS

Combination of immune checkpoint blockade with DNA cancer vaccine induces potent antitumor immunity against P815 mastocytoma

(2018) Alessandra Lopes et al.   Scientific Reports

Degradation-Regulatable Architectured Implantable Macroporous Scaffold for the Spatiotemporal Modulation of Immunosuppressive Microenvironment and Enhanced Combination Cancer Immunotherapy

(2018) Long Ren et al.   ADVANCED FUNCTIONAL MATERIALS

Bait-and-Switch Supramolecular Strategy To Generate Noncationic RNA–Polymer Complexes for RNA Delivery

(2018) Ziwen Jiang et al.   BIOMACROMOLECULES

Nanogels Enable Efficient miRNA Delivery and Target Gene Downregulation in Transfection-Resistant Multiple Myeloma Cells

(2018) Ilona Zilkowski et al.   BIOMACROMOLECULES

A bioactive collagen membrane that enhances bone regeneration

(2018) Behnoush Khorsand et al.   JOURNAL OF BIOMEDICAL MATERIALS RESEARCH PART B-APPLIED BIOMATERIALS

Biomaterials for vaccine-based cancer immunotherapy

(2018) Rui Zhang et al.   JOURNAL OF CONTROLLED RELEASE

A Dual Targeting Dendrimer-Mediated siRNA Delivery System for Effective Gene Silencing in Cancer Therapy

(2018) Yiwen Dong et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

Injectable Biomimetic Hydrogels as Tools for Efficient T Cell Expansion and Delivery

(2018) Jorieke Weiden et al.   Frontiers in Immunology

Minicircle Versus Plasmid DNA Delivery by Receptor-Targeted Polyplexes

(2017) Ana Krhac Levacic et al.   HUMAN GENE THERAPY

Transposons: Moving Forward from Preclinical Studies to Clinical Trials

(2017) Jaitip Tipanee et al.   HUMAN GENE THERAPY

Biopolymers codelivering engineered T cells and STING agonists can eliminate heterogeneous tumors

(2017) Tyrel T. Smith et al.   JOURNAL OF CLINICAL INVESTIGATION

Nucleic acid combinations: A new frontier for cancer treatment

(2017) Remant Bahadur K.C. et al.   JOURNAL OF CONTROLLED RELEASE

Translation of CircRNAs

(2017) Nagarjuna Reddy Pamudurti et al.   MOLECULAR CELL

Circ-ZNF609 Is a Circular RNA that Can Be Translated and Functions in Myogenesis

(2017) Ivano Legnini et al.   MOLECULAR CELL

Chitosan/Hyaluronic Acid Nanoparticles: Rational Design Revisited for RNA Delivery

(2017) Enrique Lallana et al.   MOLECULAR PHARMACEUTICS

Specific and Efficient Regression of Cancers Harboring KRAS Mutation by Targeted RNA Replacement

(2017) Sung Jin Kim et al.   MOLECULAR THERAPY

Improving T Cell Expansion with a Soft Touch

(2017) Lester H. Lambert et al.   NANO LETTERS

In situ programming of leukaemia-specific T cells using synthetic DNA nanocarriers

(2017) Tyrel T. Smith et al.   Nature Nanotechnology

Charge-altering releasable transporters (CARTs) for the delivery and release of mRNA in living animals

(2017) Colin J. McKinlay et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Identification of HuR target circular RNAs uncovers suppression of PABPN1 translation by CircPABPN1

(2017) Kotb Abdelmohsen et al.   RNA Biology

Modified mRNA for BMP-2 in Combination with Biomaterials Serves as a Transcript-Activated Matrix for Effectively Inducing Osteogenic Pathways in Stem Cells

(2017) Elizabeth R. Balmayor et al.   STEM CELLS AND DEVELOPMENT

Gene Therapy with the Sleeping Beauty Transposon System

(2017) Partow Kebriaei et al.   TRENDS IN GENETICS

Controlled in-cell activation of RNA therapeutics using bond-cleaving bio-orthogonal chemistry

(2017) Irfan Khan et al.   Chemical Science

Hit-and-run programming of therapeutic cytoreagents using mRNA nanocarriers

(2017) H. F. Moffett et al.   Nature Communications

Aptazyme-embedded guide RNAs enable ligand-responsive genome editing and transcriptional activation

(2017) Weixin Tang et al.   Nature Communications

Engineering CAR T Cells with Biomaterials

(2017)   Cancer Discovery

Development of Exon Skipping Therapies for Duchenne Muscular Dystrophy: A Critical Review and a Perspective on the Outstanding Issues

(2017) Annemieke Aartsma-Rus et al.   Nucleic Acid Therapeutics

Biomaterials for polynucleotide delivery to anchorage-independent cells

(2017) Aysha S. Ansari et al.   Journal of Materials Chemistry B

Advances in Non-Viral DNA Vectors for Gene Therapy

(2017) Cinnamon Hardee et al.   Genes

Global Manufacturing of CAR T Cell Therapy

(2017) Bruce L. Levine et al.   Molecular Therapy-Methods & Clinical Development

Inflammation-Triggered Cancer Immunotherapy by Programmed Delivery of CpG and Anti-PD1 Antibody

(2016) Chao Wang et al.   ADVANCED MATERIALS

Longitudinal effect of eteplirsen versus historical control on ambulation in Duchenne muscular dystrophy

(2016) Jerry R. Mendell et al.   ANNALS OF NEUROLOGY

Chemically modified RNA induces osteogenesis of stem cells and human tissue explants as well as accelerates bone healing in rats

(2016) Elizabeth R. Balmayor et al.   BIOMATERIALS

CD19-targeted CAR T-cell therapeutics for hematologic malignancies: interpreting clinical outcomes to date

(2016) Jae H. Park et al.   BLOOD

In vivo transduction of primitive mobilized hematopoietic stem cells after intravenous injection of integrating adenovirus vectors

(2016) M. Richter et al.   BLOOD

Therapeutic nucleic acids: current clinical status

(2016) Kannan Sridharan et al.   BRITISH JOURNAL OF CLINICAL PHARMACOLOGY

Induction of tumor apoptosis through a circular RNA enhancing Foxo3 activity

(2016) William W Du et al.   CELL DEATH AND DIFFERENTIATION

Effect of sustained PDGF nonviral gene delivery on repair of tooth-supporting bone defects

(2016) A B Plonka et al.   GENE THERAPY

Phase I trials using Sleeping Beauty to generate CD19-specific CAR T cells

(2016) Partow Kebriaei et al.   JOURNAL OF CLINICAL INVESTIGATION

Transcript-activated collagen matrix as sustained mRNA delivery system for bone regeneration

(2016) Zohreh Sadat Badieyan et al.   JOURNAL OF CONTROLLED RELEASE

Targeting CXCR4/SDF-1 axis by lipopolymer complexes of siRNA in acute myeloid leukemia

(2016) Breanne Landry et al.   JOURNAL OF CONTROLLED RELEASE

Enhanced CAR T-cell engineering using non-viral Sleeping Beauty transposition from minicircle vectors

(2016) R Monjezi et al.   LEUKEMIA

Viral and Synthetic RNA Vector Technologies and Applications

(2016) Juliane W Schott et al.   MOLECULAR THERAPY

Identification of senescence-associated circular RNAs (SAC-RNAs) reveals senescence suppressor CircPVT1

(2016) Amaresh C. Panda et al.   NUCLEIC ACIDS RESEARCH

A High-Capacity Adenoviral Hybrid Vector System Utilizing the Hyperactive Sleeping Beauty Transposase SB100X for Enhanced Integration

(2016) Philip Boehme et al.   Molecular Therapy-Nucleic Acids

Long-Term Efficacy, Safety, and Pharmacokinetics of Drisapersen in Duchenne Muscular Dystrophy: Results from an Open-Label Extension Study

(2016) Nathalie M. Goemans et al.   PLoS One

Self-Assembled DNA Nanoclews for the Efficient Delivery of CRISPR-Cas9 for Genome Editing

(2015) Wujin Sun et al.   ANGEWANDTE CHEMIE-INTERNATIONAL EDITION

ATP-responsive DNA-graphene hybrid nanoaggregates for anticancer drug delivery

(2015) Ran Mo et al.   BIOMATERIALS

Chemically modified RNA activated matrices enhance bone regeneration

(2015) Satheesh Elangovan et al.   JOURNAL OF CONTROLLED RELEASE

Large-scale Clinical-grade Retroviral Vector Production in a Fixed-Bed Bioreactor

(2015) Xiuyan Wang et al.   JOURNAL OF IMMUNOTHERAPY

T cell exclusion, immune privilege, and the tumor microenvironment

(2015) J. A. Joyce et al.   SCIENCE

Skin Transfection Patterns and Expression Kinetics of Electroporation-Enhanced Plasmid Delivery Using the CELLECTRA-3P, a Portable Next-Generation Dermal Electroporation Device

(2015) Dinah H. Amante et al.   Human Gene Therapy Methods

Additive nanocomplexes of cationic lipopolymers for improved non-viral gene delivery to mesenchymal stem cells

(2015) Remant Bahadur KC et al.   Journal of Materials Chemistry B

Magnetic Field-Induced T Cell Receptor Clustering by Nanoparticles Enhances T Cell Activation and Stimulates Antitumor Activity

(2014) Karlo Perica et al.   ACS Nano

Thermoreversible Poly(ethylene glycol)-g-Chitosan Hydrogel as a Therapeutic T Lymphocyte Depot for Localized Glioblastoma Immunotherapy

(2014) Ching-Ting Tsao et al.   BIOMACROMOLECULES

Impact of DNA Vector Topology on Non-Viral Gene Therapeutic Safety and Efficacy

(2014) Chi Sum et al.   CURRENT GENE THERAPY

Targeting CpG Oligonucleotides to the Lymph Node by Nanoparticles Elicits Efficient Antitumoral Immunity

(2014) C. Bourquin et al.   JOURNAL OF IMMUNOLOGY

Polymeric nanoparticle-mediated silencing of CD44 receptor in CD34+ acute myeloid leukemia cells

(2014) Hilal Gul-Uludağ et al.   LEUKEMIA RESEARCH

circRNA Biogenesis Competes with Pre-mRNA Splicing

(2014) Reut Ashwal-Fluss et al.   MOLECULAR CELL

Biopolymer implants enhance the efficacy of adoptive T-cell therapy

(2014) Sirkka B Stephan et al.   NATURE BIOTECHNOLOGY

Injectable, spontaneously assembling, inorganic scaffolds modulate immune cells in vivo and increase vaccine efficacy

(2014) Jaeyun Kim et al.   NATURE BIOTECHNOLOGY

A carbon nanotube–polymer composite for T-cell therapy

(2014) Tarek R. Fadel et al.   Nature Nanotechnology

The new frontier of genome engineering with CRISPR-Cas9

(2014) J. A. Doudna et al.   SCIENCE

Effective suppression of Dengue virus using a novel group-I intron that induces apoptotic cell death upon infection through conditional expression of the Bax C-terminal domain

(2014) James R Carter et al.   Virology Journal

DNA Ministrings: Highly Safe and Effective Gene Delivery Vectors

(2014) Nafiseh Nafissi et al.   Molecular Therapy-Nucleic Acids

Gelatin coating to stabilize the transfection ability of nucleic acid polyplexes

(2013) Laura Rose et al.   Acta Biomaterialia

Using poly(lactic-co-glycolic acid) microspheres to encapsulate plasmid of bone morphogenetic protein 2/polyethylenimine nanoparticles to promote bone formation in vitro and in vivo

(2013) Hongchen Sun et al.   International Journal of Nanomedicine

In vivo targeting of adoptively transferred T-cells with antibody- and cytokine-conjugated liposomes

(2013) Yiran Zheng et al.   JOURNAL OF CONTROLLED RELEASE

Therapeutic targeting of CD19 in hematological malignancies: past, present, future and beyond

(2013) Ben-Zion Katz et al.   LEUKEMIA & LYMPHOMA

Noncoding DNA in Lipofection of HeLa Cells—A Few Insights

(2013) Nathalie Symens et al.   MOLECULAR PHARMACEUTICS

A vector-free microfluidic platform for intracellular delivery

(2013) A. Sharei 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

ZFN, TALEN, and CRISPR/Cas-based methods for genome engineering

(2013) Thomas Gaj et al.   TRENDS IN BIOTECHNOLOGY

Cell Squeezing as a Robust, Microfluidic Intracellular Delivery Platform

(2013) Armon Sharei et al.   Jove-Journal of Visualized Experiments

A Phenylboronate-Functionalized Polyion Complex Micelle for ATP-Triggered Release of siRNA

(2012) Mitsuru Naito et al.   ANGEWANDTE CHEMIE-INTERNATIONAL EDITION

Therapeutic RNA aptamers in clinical trials

(2012) Padma Sundaram et al.   EUROPEAN JOURNAL OF PHARMACEUTICAL SCIENCES

Selective regression of cancer cells expressing a splicing variant of AIMP2 through targeted RNA replacement by trans-splicing ribozyme

(2012) You-Sub Won et al.   JOURNAL OF BIOTECHNOLOGY

Spherical Nucleic Acids

(2012) Joshua I. Cutler et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

Hollow Spherical Nucleic Acids for Intracellular Gene Regulation Based upon Biocompatible Silica Shells

(2012) Kaylie L. Young et al.   NANO LETTERS

Molecularly self-assembled nucleic acid nanoparticles for targeted in vivo siRNA delivery

(2012) Hyukjin Lee et al.   Nature Nanotechnology

Decade-Long Safety and Function of Retroviral-Modified Chimeric Antigen Receptor T Cells

(2012) J. Scholler et al.   Science Translational Medicine

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

(2011) Arjun Dhanoya et al.   JOURNAL OF BIOTECHNOLOGY

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

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

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

Interbilayer-crosslinked multilamellar vesicles as synthetic vaccines for potent humoral and cellular immune responses

(2011) James J. Moon et al.   NATURE MATERIALS

Inducible Apoptosis as a Safety Switch for Adoptive Cell Therapy

(2011) Antonio Di Stasi et al.   NEW ENGLAND JOURNAL OF MEDICINE

Rational design of a small molecule-responsive intramer controlling transgene expression in mammalian cells

(2011) David Ausländer et al.   NUCLEIC ACIDS RESEARCH

DNA Transposons: Nature and Applications in Genomics

(2010) Martin Munoz-Lopez et al.   CURRENT GENOMICS

Comparison of anti-CD3 and anti-CD28-coated beads with soluble anti-CD3 for expanding human T cells: Differing impact on CD8 T cell phenotype and responsiveness to restimulation

(2010) Yixin Li et al.   Journal of Translational Medicine

Genome editing with engineered zinc finger nucleases

(2010) Fyodor D. Urnov et al.   NATURE REVIEWS GENETICS

Design of small molecule-responsive microRNAs based on structural requirements for Drosha processing

(2010) Chase L. Beisel et al.   NUCLEIC ACIDS RESEARCH

Hydrophobic modifications of cationic polymers for gene delivery

(2010) Zonghua Liu et al.   PROGRESS IN POLYMER SCIENCE

Lipid and hydrophobic modification of cationic carriers on route to superior gene vectors

(2010) Vanessa Incani et al.   Soft Matter

Pharmacokinetics and bone formation by BMP-2 entrapped in polyethylenimine-coated albumin nanoparticles

(2009) Sufeng Zhang et al.   BIOMATERIALS

Manufacturing Validation of Biologically Functional T Cells Targeted to CD19 Antigen for Autologous Adoptive Cell Therapy

(2009) Daniel Hollyman et al.   JOURNAL OF IMMUNOTHERAPY

Transposon-mediated genome manipulation in vertebrates

(2009) Zoltán Ivics et al.   NATURE METHODS

Effects of size and topology of DNA molecules on intracellular delivery with non-viral gene carriers

(2008) Charlie YM Hsu et al.   BMC BIOTECHNOLOGY

Insertional oncogenesis in 4 patients after retrovirus-mediated gene therapy of SCID-X1

(2008) Salima Hacein-Bey-Abina et al.   JOURNAL OF CLINICAL INVESTIGATION

A Comprehensive Platform for Ex Vivo T-cell Expansion Based on Biodegradable Polymeric Artificial Antigen-presenting Cells

(2008) Erin R Steenblock et al.   MOLECULAR THERAPY

Protein production and purification

(2008) Susanne Gräslund et al.   NATURE METHODS

DNA bar coding and pyrosequencing to analyze adverse events in therapeutic gene transfer

(2008) Gary P. Wang et al.   NUCLEIC ACIDS RESEARCH

Transcriptional Activities of the Sleeping Beauty Transposon and Shielding Its Genetic Cargo With Insulators

(2007) Oliver Walisko et al.   MOLECULAR THERAPY