Plasmid DNA gene therapy of the Niemann-Pick C1 mouse with transferrin receptor-targeted Trojan horse liposomes
Published 2020 View Full Article
- Home
- Publications
- Publication Search
- Publication Details
Title
Plasmid DNA gene therapy of the Niemann-Pick C1 mouse with transferrin receptor-targeted Trojan horse liposomes
Authors
Keywords
-
Journal
Scientific Reports
Volume 10, Issue 1, Pages -
Publisher
Springer Science and Business Media LLC
Online
2020-08-07
DOI
10.1038/s41598-020-70290-w
References
Ask authors/readers for more resources
Related references
Note: Only part of the references are listed.- A therapy with miglustat, 2-hydroxypropyl-ß-cyclodextrin and allopregnanolone restores splenic cholesterol homeostasis in Niemann-pick disease type C1
- (2019) Anna-Maria Neßlauer et al. Lipids in Health and Disease
- AAV9 intracerebroventricular gene therapy improves lifespan, locomotor function and pathology in a mouse model of Niemann-Pick type C1 disease
- (2018) Michael P Hughes et al. HUMAN MOLECULAR GENETICS
- Efficient Gene Transfer to the Central Nervous System by Single-Stranded Anc80L65
- (2018) Eloise Hudry et al. Molecular Therapy-Methods & Clinical Development
- AAV9-NPC1 significantly ameliorates Purkinje cell death and behavioral abnormalities in mouse NPC disease
- (2017) Chang Xie et al. JOURNAL OF LIPID RESEARCH
- Intrathecal 2-hydroxypropyl-β-cyclodextrin decreases neurological disease progression in Niemann-Pick disease, type C1: a non-randomised, open-label, phase 1–2 trial
- (2017) Daniel S Ory et al. LANCET
- CSF, blood-brain barrier, and brain drug delivery
- (2016) William M. Pardridge Expert Opinion on Drug Delivery
- Systemic AAV9 gene therapy improves the lifespan of mice with Niemann-Pick disease, type C1
- (2016) Randy J. Chandler et al. HUMAN MOLECULAR GENETICS
- Vector design influences hepatic genotoxicity after adeno-associated virus gene therapy
- (2015) Randy J. Chandler et al. JOURNAL OF CLINICAL INVESTIGATION
- Non-invasive gene targeting to the fetal brain after intravenous administration and transplacental transfer of plasmid DNA using PEGylated immunoliposomes
- (2015) Eain M. Cornford et al. JOURNAL OF DRUG TARGETING
- Disease stage determines the efficacy of treatment of a paediatric neurodegenerative disease
- (2014) Sofia Hassiotis et al. EUROPEAN JOURNAL OF NEUROSCIENCE
- Niemann–Pick disease type C: introduction and main clinical features
- (2014) A. Burlina JOURNAL OF NEUROLOGY
- Cyclodextrin alleviates neuronal storage of cholesterol in Niemann-Pick C disease without evidence of detectable blood–brain barrier permeability
- (2013) Charles C. Pontikis et al. JOURNAL OF INHERITED METABOLIC DISEASE
- Preclinical Differences of Intravascular AAV9 Delivery to Neurons and Glia: A Comparative Study of Adult Mice and Nonhuman Primates
- (2011) Steven J Gray et al. MOLECULAR THERAPY
- Iron uptake from plasma transferrin by a transferrin receptor 2 mutant mouse model of haemochromatosis
- (2010) Anita C.G. Chua et al. JOURNAL OF HEPATOLOGY
- Weekly Cyclodextrin Administration Normalizes Cholesterol Metabolism in Nearly Every Organ of the Niemann-Pick Type C1 Mouse and Markedly Prolongs Life
- (2010) Charina M Ramirez et al. PEDIATRIC RESEARCH
- A modified protocol for efficient DNA encapsulation into pegylated immunoliposomes (PILs)
- (2009) Tina Skjørringe et al. JOURNAL OF CONTROLLED RELEASE
- Development, Validation, and Clinical Implementation of an Assay to Measure Total Antibody Response to Naglazyme® (Galsulfase)
- (2008) Joleen T. White et al. AAPS Journal
- Engineering and expression of a chimeric transferrin receptor monoclonal antibody for blood-brain barrier delivery in the mouse
- (2008) Ruben J. Boado et al. BIOTECHNOLOGY AND BIOENGINEERING
- Intravascular AAV9 preferentially targets neonatal neurons and adult astrocytes
- (2008) Kevin D Foust et al. NATURE BIOTECHNOLOGY
Find Funding. Review Successful Grants.
Explore over 25,000 new funding opportunities and over 6,000,000 successful grants.
ExplorePublish scientific posters with Peeref
Peeref publishes scientific posters from all research disciplines. Our Diamond Open Access policy means free access to content and no publication fees for authors.
Learn More