Endosomal escape cell-penetrating peptides significantly enhance pharmacological effectiveness and CNS activity of systemically administered antisense oligonucleotides

The 10th Oligonucleotide Therapy Approved: Golodirsen for Duchenne Muscular Dystrophy

(2020) Annemieke Aartsma-Rus et al.   Nucleic Acid Therapeutics

The powerful world of antisense oligonucleotides: From bench to bedside

(2020) Anaïs M. Quemener et al.   Wiley Interdisciplinary Reviews-RNA

Cell penetrating peptides: as a promising theranostics strategy to circumvent the blood-brain barrier for CNS diseases

(2020) Behrang Shiri Varnamkhasti et al.   Current Drug Delivery

Cell-Penetrating Peptides in Diagnosis and Treatment of Human Diseases: From Preclinical Research to Clinical Application

(2020) Jing Xie et al.   Frontiers in Pharmacology

Cell-Penetrating Peptides Escape the Endosome by Inducing Vesicle Budding and Collapse

(2020) Ashweta Sahni et al.   ACS Chemical Biology

Anti-α-synuclein ASO delivered to monoamine neurons prevents α-synuclein accumulation in a Parkinson's disease-like mouse model and in monkeys

(2020) Diana Alarcón-Arís et al.   EBioMedicine

Thiol-Cyanobenzothiazole Ligation for the Efficient Preparation of Peptide-PNA Conjugates

(2019) Nitin Patil et al.   BIOCONJUGATE CHEMISTRY

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

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

Randomized Trial of Verubecestat for Prodromal Alzheimer’s Disease

(2019) Michael F. Egan et al.   NEW ENGLAND JOURNAL OF MEDICINE

Novel pH Selective, Highly Lytic Peptides Based on a Chimeric Influenza Hemagglutinin Peptide/Cell Penetrating Peptide Motif

(2019) Bethany Algayer et al.   MOLECULES

Fatty acid conjugation enhances potency of antisense oligonucleotides in muscle

(2019) Thazha P Prakash et al.   NUCLEIC ACIDS RESEARCH

Lipid Conjugates Enhance Endosomal Release of Antisense Oligonucleotides Into Cells

(2019) Shiyu Wang et al.   Nucleic Acid Therapeutics

Polyhistidine facilitates direct membrane translocation of cell-penetrating peptides into cells

(2019) Han-Jung Lee et al.   Scientific Reports

Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease

(2019) Jinkuk Kim et al.   NEW ENGLAND JOURNAL OF MEDICINE

Therapeutic Antisense Targeting of Huntingtin

(2019) Anne V. Smith et al.   DNA AND CELL BIOLOGY

Nusinersen for older patients with spinal muscular atrophy: A real‐world clinical setting experience

(2019) Aravindhan Veerapandiyan et al.   MUSCLE & NERVE

The First Approved Gene Therapy Product for Cancer Ad-p53 (Gendicine): 12 Years in the Clinic

(2018) Wei-Wei Zhang et al.   HUMAN GENE THERAPY

Endosomal Escape of Antisense Oligonucleotides Internalized by Stabilin Receptors Is Regulated by Rab5C and EEA1 During Endosomal Maturation

(2018) Colton M. Miller et al.   Nucleic Acid Therapeutics

Membrane Destabilization Induced by Lipid Species Increases Activity of Phosphorothioate-Antisense Oligonucleotides

(2018) Shiyu Wang et al.   Molecular Therapy-Nucleic Acids

A novel amphipathic cell-penetrating peptide based on the N-terminal glycosaminoglycan binding region of human apolipoprotein E

(2018) Takashi Ohgita et al.   BIOCHIMICA ET BIOPHYSICA ACTA-BIOMEMBRANES

Amphiphilic lipopeptide significantly enhances uptake of charge-neutral splice switching morpholino oligonucleotide in spinal muscular atrophy patient-derived fibroblasts

(2017) Behnoosh Tajik-Ahmadabad et al.   INTERNATIONAL JOURNAL OF PHARMACEUTICS

Routes for Drug Translocation Across the Blood-Brain Barrier: Exploiting Peptides as Delivery Vectors

(2017) Mie Kristensen et al.   JOURNAL OF PHARMACEUTICAL SCIENCES

Therapies targeting DNA and RNA in Huntington's disease

(2017) Edward J Wild et al.   LANCET NEUROLOGY

A Basic ApoE-Based Peptide Mediator to Deliver Proteins across the Blood-Brain Barrier: Long-Term Efficacy, Toxicity, and Mechanism

(2017) Yu Meng et al.   MOLECULAR THERAPY

Combination of valproic acid and morpholino splice-switching oligonucleotide produces improved outcomes in spinal muscular atrophy patient-derived fibroblasts

(2017) Anna Farrelly-Rosch et al.   NEUROCHEMISTRY INTERNATIONAL

Chemistry, mechanism and clinical status of antisense oligonucleotides and duplex RNAs

(2017) Xiulong Shen et al.   NUCLEIC ACIDS RESEARCH

Transcellular vesicular transport in epithelial and endothelial cells: Challenges and opportunities

(2017) Karen Y. Y. Fung et al.   TRAFFIC

FDA Approval of Nusinersen for Spinal Muscular Atrophy Makes 2016 the Year of Splice Modulating Oligonucleotides

(2017) Annemieke Aartsma-Rus   Nucleic Acid Therapeutics

FDA Approves Eteplirsen for Duchenne Muscular Dystrophy: The Next Chapter in the Eteplirsen Saga

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

Identification of a Peptide for Systemic Brain Delivery of a Morpholino Oligonucleotide in Mouse Models of Spinal Muscular Atrophy

(2017) Fazel Shabanpoor et al.   Nucleic Acid Therapeutics

An Intrinsically Disordered Peptide Facilitates Non-Endosomal Cell Entry

(2016) Scott H. Medina et al.   ANGEWANDTE CHEMIE-INTERNATIONAL EDITION

Blood–brain barrier shuttle peptides: an emerging paradigm for brain delivery

(2016) Benjamí Oller-Salvia et al.   CHEMICAL SOCIETY REVIEWS

The delivery of therapeutic oligonucleotides

(2016) Rudolph L. Juliano   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

Trafficking of Endogenous Immunoglobulins by Endothelial Cells at the Blood-Brain Barrier

(2016) Roberto Villaseñor et al.   Scientific Reports

Delivery of therapeutic oligonucleotides with cell penetrating peptides

(2015) Prisca Boisguérin et al.   ADVANCED DRUG DELIVERY REVIEWS

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

(2015) Kariem Ezzat et al.   NANO LETTERS

An apolipoprotein E modified liposomal nanoparticle: Ligand dependent efficiency as a siRNA delivery carrier for mouse-derived brain endothelial cells

(2014) Mina Tamaru et al.   INTERNATIONAL JOURNAL OF PHARMACEUTICS

Comparison of Five Peptide Vectors for Improved Brain Delivery of the Lysosomal Enzyme Arylsulfatase A

(2014) A. Bockenhoff et al.   JOURNAL OF NEUROSCIENCE

A Myristoylated Cell-Penetrating Peptide Bearing a Transferrin Receptor-Targeting Sequence for Neuro-Targeted siRNA Delivery

(2014) Pilju Youn et al.   MOLECULAR PHARMACEUTICS

Development of a general methodology for labelling peptide–morpholino oligonucleotide conjugates using alkyne–azide click chemistry

(2013) Fazel Shabanpoor et al.   CHEMICAL COMMUNICATIONS

Influence of diverse chemical modifications on the ADME characteristics and toxicology of antisense oligonucleotides

(2013) Mehrdad Dirin et al.   EXPERT OPINION ON BIOLOGICAL THERAPY

Engineering a lysosomal enzyme with a derivative of receptor-binding domain of apoE enables delivery across the blood-brain barrier

(2013) D. Wang et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

The Uptake Mechanism of the Cell-Penetrating pVEC Peptide

(2013) Ihsan Omur Akdag et al.   Journal of Chemistry

Peptide-mediated Cell and In Vivo Delivery of Antisense Oligonucleotides and siRNA

(2012) Peter Järver et al.   Molecular Therapy-Nucleic Acids

Functionalization with ApoE-derived peptides enhances the interaction with brain capillary endothelial cells of nanoliposomes binding amyloid-beta peptide

(2011) Francesca Re et al.   JOURNAL OF BIOTECHNOLOGY

Identification of valid housekeeping genes for quantitative RT-PCR analysis of cardiosphere-derived cells preconditioned under hypoxia or with prolyl-4-hydroxylase inhibitors

(2011) Suat Cheng Tan et al.   MOLECULAR BIOLOGY REPORTS

Functionalization of liposomes with ApoE-derived peptides at different density affects cellular uptake and drug transport across a blood-brain barrier model

(2011) Francesca Re et al.   Nanomedicine-Nanotechnology Biology and Medicine

siRNA Delivery to CNS Cells using a Membrane Translocation Peptide

(2010) Marytheresa A. Ifediba et al.   BIOCONJUGATE CHEMISTRY

Delivery of Macromolecules Using Arginine-Rich Cell-Penetrating Peptides: Ways to Overcome Endosomal Entrapment

(2009) Ayman El-Sayed et al.   AAPS Journal

Gene Knockdowns in Adult Animals: PPMOs and Vivo-Morpholinos

(2009) Jon Moulton et al.   MOLECULES

Insight into the role of HSPG in the cellular uptake of apolipoprotein E-derived peptide micelles and liposomes

(2008) Eik Leupold et al.   BIOCHIMICA ET BIOPHYSICA ACTA-BIOMEMBRANES