Targeted SMN Exon Skipping: A Useful Control to Assess In Vitro and In Vivo Splice-Switching Studies

Differential uptake, kinetics and mechanisms of intracellular trafficking of next-generation antisense oligonucleotides across human cancer cell lines

(2019) Emily Linnane et al.   NUCLEIC ACIDS RESEARCH

Amido-bridged nucleic acid (AmNA)-modified antisense oligonucleotides targeting α-synuclein as a novel therapy for Parkinson’s disease

(2019) Takuya Uehara et al.   Scientific Reports

Peptide-conjugate antisense based splice-correction for Duchenne muscular dystrophy and other neuromuscular diseases

(2019) Maria K. Tsoumpra et al.   EBioMedicine

In Vitro Validation of Phosphorodiamidate Morpholino Oligomers

(2019) May T. Aung-Htut et al.   MOLECULES

Systematic Approach to Developing Splice Modulating Antisense Oligonucleotides

(2019) May Aung-Htut et al.   INTERNATIONAL JOURNAL OF MOLECULAR SCIENCES

Reduction of integrin alpha 4 activity through splice modulating antisense oligonucleotides

(2019) May T. Aung-Htut et al.   Scientific Reports

Acute hepatotoxicity of 2′ fluoro-modified 5–10–5 gapmer phosphorothioate oligonucleotides in mice correlates with intracellular protein binding and the loss of DBHS proteins

(2018) Wen Shen et al.   NUCLEIC ACIDS RESEARCH

Antisense Oligonucleotide-Mediated Terminal Intron Retention of the SMN2 Transcript

(2018) Loren L. Flynn et al.   Molecular Therapy-Nucleic Acids

A platform for discovery of functional cell-penetrating peptides for efficient multi-cargo intracellular delivery

(2018) Katrin Hoffmann et al.   Scientific Reports

Computational Tools for Splicing Defect Prediction in Breast/Ovarian Cancer Genes: How Efficient Are They at Predicting RNA Alterations?

(2018) Alejandro Moles-Fernández et al.   Frontiers in Genetics

Systematic Screening of Commonly Used Commercial Transfection Reagents towards Efficient Transfection of Single-Stranded Oligonucleotides

(2018) Tao Wang et al.   MOLECULES

Intra-endosomal trafficking mediated by lysobisphosphatidic acid contributes to intracellular release of phosphorothioate-modified antisense oligonucleotides

(2017) Shiyu Wang et al.   NUCLEIC ACIDS RESEARCH

Cell-Penetrating Peptides: From Basic Research to Clinics

(2017) Giulia Guidotti et al.   TRENDS IN PHARMACOLOGICAL SCIENCES

The Effects of 2′-O-Methoxyethyl Containing Antisense Oligonucleotides on Platelets in Human Clinical Trials

(2017) Stanley T. Crooke et al.   Nucleic Acid Therapeutics

Treatment of infantile-onset spinal muscular atrophy with nusinersen: a phase 2, open-label, dose-escalation study

(2016) Richard S Finkel et al.   LANCET

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

Morpholino-mediated SOD1 reduction ameliorates an amyotrophic lateral sclerosis disease phenotype

(2016) M. Nizzardo et al.   Scientific Reports

Delivery of therapeutic oligonucleotides with cell penetrating peptides

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

Morpholino antisense oligonucleotides targeting intronic repressor Element1 improve phenotype in SMA mouse models

(2014) Erkan Y. Osman et al.   HUMAN MOLECULAR GENETICS

Advanced morpholino oligomers: A novel approach to antiviral therapy

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

TSUNAMI: an antisense method to phenocopy splicing-associated diseases in animals

(2012) K. Sahashi et al.   GENES & DEVELOPMENT

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

Development of Therapeutic-Grade Small Interfering RNAs by Chemical Engineering

(2012) Jesper B. Bramsen et al.   Frontiers in Genetics

SpliceAid 2: A database of human splicing factors expression data and RNA target motifs

(2011) Francesco Piva et al.   HUMAN MUTATION

Rational Design of Antisense Oligomers to Induce Dystrophin Exon Skipping

(2009) Chalermchai Mitrpant et al.   MOLECULAR THERAPY

Sustained Dystrophin Expression Induced by Peptide-conjugated Morpholino Oligomers in the Muscles of mdx Mice

(2008) Natee Jearawiriyapaisarn et al.   MOLECULAR THERAPY