Supramolecular Assembly of Aminoethylene‐Lipopeptide PMO Conjugates into RNA Splice‐Switching Nanomicelles

Scavenger Receptor Class A1 Mediates Uptake of Morpholino Antisense Oligonucleotide into Dystrophic Skeletal Muscle

(2019) Shouta Miyatake et al.   Molecular Therapy-Nucleic Acids

Endosomal Size and Membrane Leakiness Influence Proton Sponge-Based Rupture of Endosomal Vesicles

(2018) Lotte M. P. Vermeulen et al.   ACS Nano

Perfluoroaryl Bicyclic Cell-Penetrating Peptides for Delivery of Antisense Oligonucleotides

(2018) Justin M. Wolfe et al.   ANGEWANDTE CHEMIE-INTERNATIONAL EDITION

Folate receptor-directed orthogonal click-functionalization of siRNA lipopolyplexes for tumor cell killing in vivo

(2018) Philipp Michael Klein et al.   BIOMATERIALS

The proton sponge hypothesis: Fable or fact?

(2018) Lotte M.P. Vermeulen et al.   EUROPEAN JOURNAL OF PHARMACEUTICS AND BIOPHARMACEUTICS

Peptide-conjugated phosphodiamidate oligomer-mediated exon skipping has benefits for cardiac function in mdx and Cmah-/-mdx mouse models of Duchenne muscular dystrophy

(2018) Alison M. Blain et al.   PLoS One

Machine Learning To Predict Cell-Penetrating Peptides for Antisense Delivery

(2018) Justin M. Wolfe et al.   ACS Central Science

Novel peptide-dendrimer/lipid/oligonucleotide ternary complexes for efficient cellular uptake and improved splice-switching activity

(2018) Osama Saher et al.   EUROPEAN JOURNAL OF PHARMACEUTICS AND BIOPHARMACEUTICS

A Single Methylene Group in Oligoalkylamine-Based Cationic Polymers and Lipids Promotes Enhanced mRNA Delivery

(2016) Anita Jarzębińska et al.   ANGEWANDTE CHEMIE-INTERNATIONAL EDITION

Combinatorial Optimization of Sequence-Defined Oligo(ethanamino)amides for Folate Receptor-Targeted pDNA and siRNA Delivery

(2016) Dongsheng He et al.   BIOCONJUGATE CHEMISTRY

Acid-labile pHPMA modification of four-arm oligoaminoamide pDNA polyplexes balances shielding and gene transfer activity in vitro and in vivo

(2016) Linda Beckert et al.   EUROPEAN JOURNAL OF PHARMACEUTICS AND BIOPHARMACEUTICS

A novel tyrosine-modified low molecular weight polyethylenimine (P10Y) for efficient siRNA delivery in vitro and in vivo

(2016) Alexander Ewe et al.   JOURNAL OF CONTROLLED RELEASE

Precise redox-sensitive cleavage sites for improved bioactivity of siRNA lipopolyplexes

(2016) Philipp Michael Klein et al.   Nanoscale

Splice-switching antisense oligonucleotides as therapeutic drugs

(2016) Mallory A. Havens et al.   NUCLEIC ACIDS RESEARCH

Systemic peptide-mediated oligonucleotide therapy improves long-term survival in spinal muscular atrophy

(2016) Suzan M. Hammond et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Four Novel Splice-Switch Reporter Cell Lines: Distinct Impact of Oligonucleotide Chemistry and Delivery Vector on Biological Activity

(2016) Cristina S.J. Rocha et al.   Nucleic Acid Therapeutics

Nucleic Acid Therapeutics Using Polyplexes: A Journey of 50 Years (and Beyond)

(2015) Ulrich Lächelt et al.   CHEMICAL REVIEWS

Self-Assembly into Nanoparticles Is Essential for Receptor Mediated Uptake of Therapeutic Antisense Oligonucleotides

(2015) Kariem Ezzat et al.   NANO LETTERS

Histidine-rich stabilized polyplexes for cMet-directed tumor-targeted gene transfer

(2015) Petra Kos et al.   Nanoscale

Prevention of exercised induced cardiomyopathy following Pip-PMO treatment in dystrophic mdx mice

(2015) Corinne A. Betts et al.   Scientific Reports

Intravitreal Injection of Splice-switching Oligonucleotides to Manipulate Splicing in Retinal Cells

(2015) Xavier Gérard et al.   Molecular Therapy-Nucleic Acids

Modulated Protonation of Side Chain Aminoethylene Repeats in N-Substituted Polyaspartamides Promotes mRNA Transfection

(2014) Hirokuni Uchida et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

Fundamental Molecular Mechanism for the Cellular Uptake of Guanidinium-Rich Molecules

(2014) Henry D. Herce et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

Comb-Like Oligoaminoethane Carriers: Change in Topology Improves pDNA Delivery

(2013) Claudia Scholz et al.   BIOCONJUGATE CHEMISTRY

Fine-tuning of proton sponges by precise diaminoethanes and histidines in pDNA polyplexes

(2013) Ulrich Lächelt et al.   Nanomedicine-Nanotechnology Biology and Medicine

Advanced morpholino oligomers: A novel approach to antiviral therapy

(2012) Travis K. Warren et al.   ANTIVIRAL RESEARCH

Optimization of Brush-Like Cationic Copolymers for Nonviral Gene Delivery

(2012) Hua Wei et al.   BIOMACROMOLECULES

Stabilizing effect of tyrosine trimers on pDNA and siRNA polyplexes

(2012) Christina Troiber et al.   BIOMATERIALS

Structure–activity relationships of siRNA carriers based on sequence-defined oligo (ethane amino) amides

(2012) Thomas Fröhlich et al.   JOURNAL OF CONTROLLED RELEASE

RNA therapeutics: beyond RNA interference and antisense oligonucleotides

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

Bodywide skipping of exons 45-55 in dystrophic mdx52 mice by systemic antisense delivery

(2012) Y. Aoki et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Pip6-PMO, A New Generation of Peptide-oligonucleotide Conjugates With Improved Cardiac Exon Skipping Activity for DMD Treatment

(2012) Corinne Betts et al.   Molecular Therapy-Nucleic Acids

Solid-Phase Synthesis of Sequence-Defined T-, i-, and U-Shape Polymers for pDNA and siRNA Delivery

(2011) David Schaffert et al.   ANGEWANDTE CHEMIE-INTERNATIONAL EDITION

Odd–Even Effect of Repeating Aminoethylene Units in the Side Chain of N-Substituted Polyaspartamides on Gene Transfection Profiles

(2011) Hirokuni Uchida et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

Targeting RNA to treat neuromuscular disease

(2011) Francesco Muntoni et al.   NATURE REVIEWS DRUG DISCOVERY

Novel Fmoc-Polyamino Acids for Solid-Phase Synthesis of Defined Polyamidoamines

(2011) David Schaffert et al.   ORGANIC LETTERS

Antitumorigenic potential of STAT3 alternative splicing modulation

(2011) F. Zammarchi et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Translocation of HIV TAT peptide and analogues induced by multiplexed membrane and cytoskeletal interactions

(2011) A. Mishra et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Backbone rigidity and static presentation of guanidinium groups increases cellular uptake of arginine-rich cell-penetrating peptides

(2011) Gisela Lättig-Tünnemann et al.   Nature Communications

Proton Sponge Trick for pH-Sensitive Disassembly of Polyethylenimine-Based siRNA Delivery Systems

(2010) Gaelle Creusat et al.   BIOCONJUGATE CHEMISTRY

Therapeutic Potential of Splice-Switching Oligonucleotides

(2009) John Bauman et al.   OLIGONUCLEOTIDES

RNA repair restores hemoglobin expression in IVS2-654 thalassemic mice

(2009) S. Svasti et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Vivo-Morpholinos: A non-peptide transporter delivers Morpholinos into a wide array of mouse tissues

(2008) Paul A. Morcos et al.   BIOTECHNIQUES

Self-Assembling Polyethylenimine Derivatives Mediate Efficient siRNA Delivery in Mammalian Cells

(2008) Gaelle Creusat et al.   CHEMBIOCHEM

Oligoethylenimine-grafted polypropylenimine dendrimers as degradable and biocompatible synthetic vectors for gene delivery

(2008) Verena Russ et al.   JOURNAL OF CONTROLLED RELEASE

Polyplexes from Poly(aspartamide) Bearing 1,2-Diaminoethane Side Chains Induce pH-Selective, Endosomal Membrane Destabilization with Amplified Transfection and Negligible Cytotoxicity

(2008) Kanjiro Miyata et al.   JOURNAL OF THE AMERICAN CHEMICAL SOCIETY

An Endogenous TNF-α Antagonist Induced by Splice-switching Oligonucleotides Reduces Inflammation in Hepatitis and Arthritis Mouse Models

(2008) Maria A Graziewicz et al.   MOLECULAR THERAPY

Cell penetrating peptide conjugates of steric block oligonucleotides

(2007) Bernard Lebleu et al.   ADVANCED DRUG DELIVERY REVIEWS

Bioreducible poly(amido amine)s with oligoamine side chains: Synthesis, characterization, and structural effects on gene delivery

(2007) Chao Lin et al.   JOURNAL OF CONTROLLED RELEASE