Polyethylene glycol-b-poly(lactic acid) polymersomes as vehicles for enzyme replacement therapy

Emerging therapies for neuropathic lysosomal storage disorders

(2017) Jessica M. Kelly et al.   PROGRESS IN NEUROBIOLOGY

Enzyme replacement for GM1-gangliosidosis: Uptake, lysosomal activation, and cellular disease correction using a novel β-galactosidase:RTB lectin fusion

(2016) Jose Condori et al.   MOLECULAR GENETICS AND METABOLISM

Protein adsorption is required for stealth effect of poly(ethylene glycol)- and poly(phosphoester)-coated nanocarriers

(2016) Susanne Schöttler et al.   Nature Nanotechnology

Lyoprotectants modify and stabilize self-assembly of polymersomes

(2016) Jessica M. Kelly et al.   POLYMER

Multifaceted polymersome platforms: Spanning from self-assembly to drug delivery and protocells

(2016) Vimalkumar Balasubramanian et al.   PROGRESS IN POLYMER SCIENCE

Polymer-based nanoparticles for protein delivery: design, strategies and applications

(2016) Hong Zhao et al.   Journal of Materials Chemistry B

Targeted Polymeric Nanoparticles for Brain Delivery of High Molecular Weight Molecules in Lysosomal Storage Disorders

(2016) Marika Salvalaio et al.   PLoS One

Systemic AAV9 gene transfer in adult GM1 gangliosidosis mice reduces lysosomal storage in CNS and extends lifespan

(2015) Cara M. Weismann et al.   HUMAN MOLECULAR GENETICS

Biomarkers of central nervous system inflammation in infantile and juvenile gangliosidoses

(2015) Jeanine R. Jarnes Utz et al.   MOLECULAR GENETICS AND METABOLISM

Recent Advances in Delivery Through the Blood-Brain Barrier

(2014) Jessica Larsen et al.   CURRENT TOPICS IN MEDICINAL CHEMISTRY

Comparison of Five Peptide Vectors for Improved Brain Delivery of the Lysosomal Enzyme Arylsulfatase A

(2014) A. Bockenhoff et al.   JOURNAL OF NEUROSCIENCE

Delivery of an enzyme-IGFII fusion protein to the mouse brain is therapeutic for mucopolysaccharidosis type IIIB

(2014) S.-h. Kan et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Gangliosides and Gangliosidoses: Principles of Molecular and Metabolic Pathogenesis

(2013) K. Sandhoff et al.   JOURNAL OF NEUROSCIENCE

High loading of hydrophilic/hydrophobic doxorubicin into polyphosphazene polymersome for breast cancer therapy

(2013) Jing Xu et al.   Nanomedicine-Nanotechnology Biology and Medicine

Engineering a lysosomal enzyme with a derivative of receptor-binding domain of apoE enables delivery across the blood-brain barrier

(2013) D. Wang et al.   PROCEEDINGS OF THE NATIONAL ACADEMY OF SCIENCES OF THE UNITED STATES OF AMERICA

Enzyme replacement therapy for lysosomal storage diseases

(2011) Robin H. Lachmann   CURRENT OPINION IN PEDIATRICS

Gene therapy for lysosomal storage disorders

(2011) Angela Gritti   EXPERT OPINION ON BIOLOGICAL THERAPY

Crystal Structure of Human β-Galactosidase

(2011) Umeharu Ohto et al.   JOURNAL OF BIOLOGICAL CHEMISTRY

Doxorubicin-Loaded (PEG)3-PLA Nanopolymersomes: Effect of Solvents and Process Parameters on Formulation Development andIn VitroStudy

(2011) Wubeante Yenet Ayen et al.   MOLECULAR PHARMACEUTICS

New biotechnological and nanomedicine strategies for treatment of lysosomal storage disorders

(2010) Silvia Muro   Wiley Interdisciplinary Reviews-Nanomedicine and Nanobiotechnology

Stimuli-Responsive Polymersomes for Programmed Drug Delivery

(2009) Fenghua Meng et al.   BIOMACROMOLECULES

Preparation and brain delivery property of biodegradable polymersomes conjugated with OX26

(2008) Zhiqing Pang et al.   JOURNAL OF CONTROLLED RELEASE

Stimuli-Responsive Polymersomes as Nanocarriers for Drug and Gene Delivery

(2008) Ozana Onaca et al.   MACROMOLECULAR BIOSCIENCE

Generation and characterization of recombinant feline β-galactosidase for preclinical enzyme replacement therapy studies in GM1 gangliosidosis

(2008) Tatiana I. Samoylova et al.   METABOLIC BRAIN DISEASE

Molecular consequences of the pathogenic mutation in feline GM1 gangliosidosis

(2008) Douglas R. Martin et al.   MOLECULAR GENETICS AND METABOLISM