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Modified h CFTR mRNA restores normal lung function in a mouse model of cystic fibrosis

https://doi.org/10.1101/202853

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What this work says

Outgoing claims: the papers this work cites, builds on, or argues with.

inferredcites →https://doi.org/10.1002/anie.201608450openalex-snapshot

Polymer–Lipid Nanoparticles for Systemic Delivery of mRNA to the Lungs

inferredcites →https://doi.org/10.1002/cyto.a.22290openalex-snapshot

A sensitive tool to measure CFTR channel activity

inferredcites →https://doi.org/10.1016/j.bios.2008.07.051openalex-snapshot

Early determination of cystic fibrosis by electrochemical chloride quantification in sweat

inferredcites →https://doi.org/10.1016/j.immuni.2005.06.008openalex-snapshot

Suppression of RNA Recognition by Toll-like Receptors: The Impact of Nucleoside Modification and the Evolutionary Origin of RNA

inferredcites →https://doi.org/10.1016/j.jcf.2009.03.002openalex-snapshot

Health related quality of life in cystic fibrosis: To work or not to work?

inferredcites →https://doi.org/10.1016/j.jcf.2013.05.001openalex-snapshot

Modification of the salivary secretion assay in F508del mice — The murine equivalent of the human sweat test

inferredcites →https://doi.org/10.1016/j.jcf.2016.05.002openalex-snapshot

Early pulmonary disease manifestations in cystic fibrosis mice

inferredcites →https://doi.org/10.1016/j.jconrel.2015.08.007openalex-snapshot

Expression kinetics of nucleoside-modified mRNA delivered in lipid nanoparticles to mice by various routes

inferredcites →https://doi.org/10.1016/j.jconrel.2015.08.051openalex-snapshot

N1-methylpseudouridine-incorporated mRNA outperforms pseudouridine-incorporated mRNA by providing enhanced protein expression and reduced immunogenicity in mammalian cell lines and mice

inferredcites →https://doi.org/10.1016/j.pharmthera.2014.06.005openalex-snapshot

New pharmacological approaches for cystic fibrosis: Promises, progress, pitfalls

inferredcites →https://doi.org/10.1016/s0092-8674(00)81724-9openalex-snapshot

Evidence for Periciliary Liquid Layer Depletion, Not Abnormal Ion Composition, in the Pathogenesis of Cystic Fibrosis Airways Disease

inferredcites →https://doi.org/10.1038/mt.2008.200openalex-snapshot

Incorporation of Pseudouridine Into mRNA Yields Superior Nonimmunogenic Vector With Increased Translational Capacity and Biological Stability

inferredcites →https://doi.org/10.1038/nature06013openalex-snapshot

DAI (DLM-1/ZBP1) is a cytosolic DNA sensor and an activator of innate immune response

inferredcites →https://doi.org/10.1038/nbt.1733openalex-snapshot

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

inferredcites →https://doi.org/10.1038/nbt.2682openalex-snapshot

Modified mRNA directs the fate of heart progenitor cells and induces vascular regeneration after myocardial infarction

inferredcites →https://doi.org/10.1038/nbt.3241openalex-snapshot

In vivo genome editing using nuclease-encoding mRNA corrects SP-B deficiency

inferredcites →https://doi.org/10.1038/ncomms14630openalex-snapshot

Administration of nucleoside-modified mRNA encoding broadly neutralizing antibody protects humanized mice from HIV-1 challenge

inferredcites →https://doi.org/10.1038/nm.2715openalex-snapshot

Cystic fibrosis: a mucosal immunodeficiency syndrome

inferredcites →https://doi.org/10.1038/nrd4278openalex-snapshot

mRNA-based therapeutics — developing a new class of drugs

inferredcites →https://doi.org/10.1038/nrg3849openalex-snapshot

Cystic fibrosis genetics: from molecular understanding to clinical application

inferredcites →https://doi.org/10.1038/sj.gt.3302368openalex-snapshot

Gene therapy for cystic fibrosis: an example for lung gene therapy

inferredcites →https://doi.org/10.1038/sj.gt.3302766openalex-snapshot

Long-term expression and repeated administration of AAV type 1, 2 and 5 vectors in skeletal muscle of immunocompetent adult mice

inferredcites →https://doi.org/10.1056/nejm199204303261804openalex-snapshot

Prediction of Mortality in Patients with Cystic Fibrosis

inferredcites →https://doi.org/10.1056/nejmoa1105185openalex-snapshot

A CFTR Potentiator in Patients with Cystic Fibrosis and the G551D Mutation

inferredcites →https://doi.org/10.1073/pnas.0904709106openalex-snapshot

Rescue of CF airway epithelial cell function in vitro by a CFTR potentiator, VX-770

inferredcites →https://doi.org/10.1080/019021400750048090openalex-snapshot

DOES LACK OF Cftr GENE LEAD TO DEVELOPMENTAL ABNORMALITIES IN THE LUNG?

inferredcites →https://doi.org/10.1080/23808993.2016.1175299openalex-snapshot

Efficacy of lumacaftor-ivacaftor for the treatment of cystic fibrosis patients homozygous for the F508del-CFTR mutation

inferredcites →https://doi.org/10.1089/10430349950016384openalex-snapshot

A Phase I Study of Adenovirus-Mediated Transfer of the Human Cystic Fibrosis Transmembrane Conductance Regulator Gene to a Lung Segment of Individuals with Cystic Fibrosis

inferredcites →https://doi.org/10.1089/hum.1997.8.1-15openalex-snapshot

Aerosol Administration of a Recombinant Adenovirus Expressing CFTR to Cystic Fibrosis Patients: A Phase I Clinical Trial

inferredcites →https://doi.org/10.1089/jamp.2010.0836openalex-snapshot

The Particle has Landed—Characterizing the Fate of Inhaled Pharmaceuticals

inferredcites →https://doi.org/10.1093/nar/gkq347openalex-snapshot

Incorporation of pseudouridine into mRNA enhances translation by diminishing PKR activation

inferredcites →https://doi.org/10.1126/science.2475911openalex-snapshot

Identification of the Cystic Fibrosis Gene: Cloning and Characterization of Complementary DNA

inferredcites →https://doi.org/10.1126/science.2570460openalex-snapshot

Identification of the Cystic Fibrosis Gene: Genetic Analysis

inferredcites →https://doi.org/10.1136/thoraxjnl-2011-200393openalex-snapshot

Results of a phase IIa study of VX-809, an investigational CFTR corrector compound, in subjects with cystic fibrosis homozygous for the F508del-CFTR mutation

inferredcites →https://doi.org/10.1146/annurev.med.58.071905.105316openalex-snapshot

Airway Surface Dehydration in Cystic Fibrosis: Pathogenesis and Therapy

inferredcites →https://doi.org/10.1152/ajpcell.2001.281.5.c1734openalex-snapshot

Cell-based assay for high-throughput quantitative screening of CFTR chloride transport agonists

inferredcites →https://doi.org/10.1172/jci119861openalex-snapshot

Lung disease in mice with cystic fibrosis.

inferredcites →https://doi.org/10.1172/jci65351openalex-snapshot

Modified Foxp3 mRNA protects against asthma through an IL-10–dependent mechanism

inferredcites →https://doi.org/10.1186/1471-2156-5-21openalex-snapshot

The "Goldilocks Effect" in Cystic Fibrosis: identification of a lung phenotype in the cftr knockout and heterozygous mouse

inferredcites →https://doi.org/10.1186/1479-0556-2-9openalex-snapshot

The use of retroviral vectors for gene therapy-what are the risks? A review of retroviral pathogenesis and its relevance to retroviral vector-mediated gene delivery

inferredcites →https://doi.org/10.1186/s40348-015-0022-6openalex-snapshot

Modified mRNA as a new therapeutic option for pediatric respiratory diseases and hemoglobinopathies

inferredcites →https://doi.org/10.1371/journal.pone.0071057openalex-snapshot

A Human In Vitro Whole Blood Assay to Predict the Systemic Cytokine Response to Therapeutic Oligonucleotides Including siRNA

inferredcites →https://doi.org/10.1373/clinchem.2008.112797openalex-snapshot

The MIQE Guidelines: Minimum Information for Publication of Quantitative Real-Time PCR Experiments

inferredcites →https://doi.org/10.1513/pats.200703-043bropenalex-snapshot

Detection of Cystic Fibrosis Transmembrane Conductance Regulator Activity in Early-Phase Clinical Trials

inferredcites →https://doi.org/10.1590/s1806-37132011000200008openalex-snapshot

Avaliação da qualidade de vida de pacientes com fibrose cística por meio do Cystic Fibrosis Questionnaire

inferredcites →https://doi.org/10.18632/oncotarget.231openalex-snapshot

The Challenge for Gene Therapy: Innate Immune Response to Adenoviruses

inferredcites →https://doi.org/10.3390/ijms15045852openalex-snapshot

Nanoparticle-Mediated Pulmonary Drug Delivery: A Review

inferredcites →https://doi.org/10.7326/m13-0636openalex-snapshot

Longevity of Patients With Cystic Fibrosis in 2000 to 2010 and Beyond: Survival Analysis of the Cystic Fibrosis Foundation Patient Registry

What points at this work

Incoming claims: where this work appears in other papers’ reference lists and arguments.

inferred← citeshttps://doi.org/10.1016/j.nano.2019.102073openalex-snapshot

Preferential uptake of chitosan-coated PLGA nanoparticles by primary human antigen presenting cells

inferred← citeshttps://doi.org/10.1016/j.ymthe.2018.05.014openalex-snapshot

Lipid Nanoparticle-Delivered Chemically Modified mRNA Restores Chloride Secretion in Cystic Fibrosis

inferred← citeshttps://doi.org/10.1016/j.ymthe.2019.02.012openalex-snapshot

Delivering the Messenger: Advances in Technologies for Therapeutic mRNA Delivery

inferred← citeshttps://doi.org/10.1038/s41434-022-00379-6openalex-snapshot

Empowering gene delivery with protein engineering platforms

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