Delivery Characterization of SPL84 Inhaled Antisense Oligonucleotide Drug for 3849 + 10 kb C- > T Cystic Fibrosis Patients

Physiology and Pathophysiology of Human Airway Mucus

(2022) David B. Hill et al.   PHYSIOLOGICAL REVIEWS

Delivery of oligonucleotide‐based therapeutics: challenges and opportunities

(2021) Suzan M Hammond et al.   EMBO Molecular Medicine

Lipid Nanoparticles—From Liposomes to mRNA Vaccine Delivery, a Landscape of Research Diversity and Advancement

(2021) Rumiana Tenchov et al.   ACS Nano

Antisense oligonucleotide-based drug development for Cystic Fibrosis patients carrying the 3849+10 kb C-to-T splicing mutation

(2021) Yifat S. Oren et al.   Journal of Cystic Fibrosis

Efficient Delivery of Antisense Oligonucleotides Using Bioreducible Lipid Nanoparticles In Vitro and In Vivo

(2020) Liu Yang et al.   Molecular Therapy-Nucleic Acids

Optimization of antisense-mediated exon skipping for Duchenne muscular dystrophy

(2020) Kasia Dzierlega et al.   GENE THERAPY

Insights on animal models to investigate inhalation therapy: Relevance for biotherapeutics

(2018) A. Guillon et al.   INTERNATIONAL JOURNAL OF PHARMACEUTICS

CFTR modulator theratyping: Current status, gaps and future directions

(2018) John Paul Clancy et al.   Journal of Cystic Fibrosis

Advances in therapy for spinal muscular atrophy: promises and challenges

(2018) Ewout J. N. Groen et al.   Nature Reviews Neurology

Nusinersen versus Sham Control in Later-Onset Spinal Muscular Atrophy

(2018) Eugenio Mercuri et al.   NEW ENGLAND JOURNAL OF MEDICINE

Intracellular Trafficking and Endosomal Release of Oligonucleotides: What We Know and What We Don’t

(2018) R.L. Juliano   Nucleic Acid Therapeutics

Pathological Mucus and Impaired Mucus Clearance in Cystic Fibrosis Patients Results from Increased Concentration, not altered pH

(2018) David B. Hill et al.   EUROPEAN RESPIRATORY JOURNAL

Inhaled ENaC antisense oligonucleotide ameliorates cystic fibrosis-like lung disease in mice

(2017) Jeff R. Crosby et al.   Journal of Cystic Fibrosis

The chemical evolution of oligonucleotide therapies of clinical utility

(2017) Anastasia Khvorova et al.   NATURE BIOTECHNOLOGY

Correction of CFTR function in nasal epithelial cells from cystic fibrosis patients predicts improvement of respiratory function by CFTR modulators

(2017) Iwona M. Pranke et al.   Scientific Reports

WS18.6 QR-010 penetrates the CF-like mucus barrier in vitro and in vivo

(2016) V. Brinks et al.   Journal of Cystic Fibrosis

Toward the modeling of mucus draining from human lung: role of airways deformation on air-mucus interaction

(2015) Benjamin Mauroy et al.   Frontiers in Physiology

Local and systemic tolerability of a 2′O-methoxyethyl antisense oligonucleotide targeting interleukin-4 receptor-α delivery by inhalation in mouse and monkey

(2014) Robert A. Fey et al.   INHALATION TOXICOLOGY

Antisense oligonucleotides: modifications and clinical trials

(2014) Vivek K. Sharma et al.   MedChemComm

Comparison of Cystic Fibrosis Transmembrane Conductance Regulator (CFTR) and Ciliary Beat Frequency Activation by the CFTR Modulators Genistein, VRT-532, and UCCF-152 in Primary Sinonasal Epithelial Cultures

(2014) Bryant T. Conger et al.   JAMA Otolaryngology-Head & Neck Surgery

RNA therapeutics: beyond RNA interference and antisense oligonucleotides

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

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

(2010) C. Frank Bennett et al.   Annual Review of Pharmacology and Toxicology

Antisense correction of SMN2 splicing in the CNS rescues necrosis in a type III SMA mouse model

(2010) Y. Hua et al.   GENES & DEVELOPMENT