Self-assembled peptide–poloxamine nanoparticles enable in vitro and in vivo genome restoration for cystic fibrosis
出版年份 2019 全文链接
标题
Self-assembled peptide–poloxamine nanoparticles enable in vitro and in vivo genome restoration for cystic fibrosis
作者
关键词
-
出版物
Nature Nanotechnology
Volume 14, Issue 3, Pages 287-297
出版商
Springer Nature
发表日期
2019-01-29
DOI
10.1038/s41565-018-0358-x
参考文献
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注意:仅列出部分参考文献,下载原文获取全部文献信息。- PEGylation for enhancing nanoparticle diffusion in mucus
- (2018) Justin T. Huckaby et al. ADVANCED DRUG DELIVERY REVIEWS
- Animal and model systems for studying cystic fibrosis
- (2018) Bradley H. Rosen et al. Journal of Cystic Fibrosis
- Overcoming cellular barriers for RNA therapeutics
- (2017) Steven F Dowdy NATURE BIOTECHNOLOGY
- Delivery technologies for genome editing
- (2017) Hao Yin et al. NATURE REVIEWS DRUG DISCOVERY
- Gene Therapy with the Sleeping Beauty Transposon System
- (2017) Partow Kebriaei et al. TRENDS IN GENETICS
- Barriers to inhaled gene therapy of obstructive lung diseases: A review
- (2016) Namho Kim et al. JOURNAL OF CONTROLLED RELEASE
- Cystic fibrosis
- (2016) J Stuart Elborn LANCET
- The Mucus Barrier to Inhaled Gene Therapy
- (2016) Gregg A Duncan et al. MOLECULAR THERAPY
- In vitro and ex vivo strategies for intracellular delivery
- (2016) Martin P. Stewart et al. NATURE
- Preparation for a first-in-man lentivirus trial in patients with cystic fibrosis
- (2016) Eric W F W Alton et al. THORAX
- Evaluation of tetrafunctional block copolymers as synthetic vectors for lung gene transfer
- (2015) Peggy Richard-Fiardo et al. BIOMATERIALS
- Hybrid Nonviral/Viral Vector Systems for Improved piggyBac DNA Transposon In Vivo Delivery
- (2015) Ashley L Cooney et al. MOLECULAR THERAPY
- Principles of nanoparticle design for overcoming biological barriers to drug delivery
- (2015) Elvin Blanco et al. NATURE BIOTECHNOLOGY
- Therapeutic genome editing: prospects and challenges
- (2015) David Benjamin Turitz Cox et al. NATURE MEDICINE
- Origins of Cystic Fibrosis Lung Disease
- (2015) David A. Stoltz et al. NEW ENGLAND JOURNAL OF MEDICINE
- Nebulisation of IVT mRNA Complexes for Intrapulmonary Administration
- (2015) Sarah M. Johler et al. PLoS One
- Highly compacted biodegradable DNA nanoparticles capable of overcoming the mucus barrier for inhaled lung gene therapy
- (2015) Panagiotis Mastorakos et al. PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA
- Nanocomplexes for gene therapy of respiratory diseases: Targeting and overcoming the mucus barrier
- (2015) Sante Di Gioia et al. PULMONARY PHARMACOLOGY & THERAPEUTICS
- Nanoparticles that deliver triplex-forming peptide nucleic acid molecules correct F508del CFTR in airway epithelium
- (2015) Nicole Ali McNeer et al. Nature Communications
- Repeated nebulisation of non-viral CFTR gene therapy in patients with cystic fibrosis: a randomised, double-blind, placebo-controlled, phase 2b trial
- (2015) Eric W F W Alton et al. Lancet Respiratory Medicine
- Lung gene therapy with highly compacted DNA nanoparticles that overcome the mucus barrier
- (2014) Jung Soo Suk et al. JOURNAL OF CONTROLLED RELEASE
- Antibiotic-free nanotherapeutics: Ultra-small, mucus-penetrating solid lipid nanoparticles enhance the pulmonary delivery and anti-virulence efficacy of novel quorum sensing inhibitors
- (2014) Noha Nafee et al. JOURNAL OF CONTROLLED RELEASE
- Cationic lipid-mediated delivery of proteins enables efficient protein-based genome editing in vitro and in vivo
- (2014) John A Zuris et al. NATURE BIOTECHNOLOGY
- In vivo endothelial siRNA delivery using polymeric nanoparticles with low molecular weight
- (2014) James E. Dahlman et al. Nature Nanotechnology
- Non-viral vectors for gene-based therapy
- (2014) Hao Yin et al. NATURE REVIEWS GENETICS
- Functional Repair of CFTR by CRISPR/Cas9 in Intestinal Stem Cell Organoids of Cystic Fibrosis Patients
- (2013) Gerald Schwank et al. Cell Stem Cell
- A functional CFTR assay using primary cystic fibrosis intestinal organoids
- (2013) Johanna F Dekkers et al. NATURE MEDICINE
- Retargeting Sleeping Beauty Transposon Insertions by Engineered Zinc Finger DNA-binding Domains
- (2012) Katrin Voigt et al. MOLECULAR THERAPY
- Biodegradable Nanoparticles Composed Entirely of Safe Materials that Rapidly Penetrate Human Mucus
- (2011) Ming Yang et al. ANGEWANDTE CHEMIE-INTERNATIONAL EDITION
- Receptor-targeted liposome-peptide nanocomplexes for siRNA delivery
- (2011) Aristides D. Tagalakis et al. BIOMATERIALS
- Expression of therapeutic proteins after delivery of chemically modified mRNA in mice
- (2011) Michael S D Kormann et al. NATURE BIOTECHNOLOGY
- The penetration of fresh undiluted sputum expectorated by cystic fibrosis patients by non-adhesive polymer nanoparticles
- (2009) Jung Soo Suk et al. BIOMATERIALS
- Parameters affecting organization and transfection efficiency of amphiphilic copolymers/DNA carriers
- (2009) Caroline Roques et al. JOURNAL OF CONTROLLED RELEASE
- Disruption of the CFTR Gene Produces a Model of Cystic Fibrosis in Newborn Pigs
- (2008) C. S. Rogers et al. SCIENCE
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