Nanotechnology‐based approaches for treating lysosomal storage disorders, a focus on Fabry disease

Extracellular vesicles increase the enzymatic activity of lysosomal proteins and improve the efficacy of enzyme replacement therapy in Fabry disease

(2020) Ibane Abasolo et al.   MOLECULAR GENETICS AND METABOLISM

Bacterial Inclusion Bodies: A Treasure Trove of Bioactive Proteins

(2020) Priyank Singhvi et al.   TRENDS IN BIOTECHNOLOGY

Brain delivery and activity of a lysosomal enzyme using a blood-brain barrier transport vehicle in mice

(2020) Julie C. Ullman et al.   Science Translational Medicine

Analysis of Renal and Cardiac Outcomes in Male Participants in the Fabry Outcome Survey Starting Agalsidase Alfa Enzyme Replacement Therapy Before and After 18 Years of Age

(2020) Rossella Parini et al.   Drug Design Development and Therapy

A Brief Reminder of Systems of Production and Chromatography-Based Recovery of Recombinant Protein Biopharmaceuticals

(2019) B. Owczarek et al.   Biomed Research International

Exosome-mediated delivery of active glucocerebrosidase to Gaucher model cells

(2019) Hojun Choi et al.   MOLECULAR GENETICS AND METABOLISM

Targeted nanoliposomes for the treatment of Fabry disease

(2019) Ibane Abasolo et al.   MOLECULAR GENETICS AND METABOLISM

FACTs Fabry gene therapy clinical trial: Two-year data

(2019) Jeffrey A. Medin et al.   MOLECULAR GENETICS AND METABOLISM

Pegunigalsidase alfa, a novel PEGylated enzyme replacement therapy for Fabry disease, provides sustained plasma concentrations and favorable pharmacodynamics: a 1-year Phase 1/2 clinical trial

(2019) Raphael Schiffmann et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Pharmacokinetics, pharmacodynamics, and safety of moss-aGalactosidase A in patients with Fabry disease

(2019) Julia B. Hennermann et al.   JOURNAL OF INHERITED METABOLIC DISEASE

The glycosylation design space for recombinant lysosomal replacement enzymes produced in CHO cells

(2019) Weihua Tian et al.   Nature Communications

TPP1 Delivery to Lysosomes with Extracellular Vesicles and their Enhanced Brain Distribution in the Animal Model of Batten Disease

(2019) Matthew J. Haney et al.   Advanced Healthcare Materials

Targeting Antitumoral Proteins to Breast Cancer by Local Administration of Functional Inclusion Bodies

(2019) Mireia Pesarrodona et al.   Advanced Science

Targeted delivery of lysosomal enzymes to the endocytic compartment in human cells using engineered extracellular vesicles

(2019) Mai Anh Do et al.   Scientific Reports

Release of targeted protein nanoparticles from functional bacterial amyloids: A death star-like approach

(2018) Ugutz Unzueta et al.   JOURNAL OF CONTROLLED RELEASE

A Blood-Brain-Barrier-Penetrating Anti-human Transferrin Receptor Antibody Fusion Protein for Neuronopathic Mucopolysaccharidosis II

(2018) Hiroyuki Sonoda et al.   MOLECULAR THERAPY

The New York pilot newborn screening program for lysosomal storage diseases: Report of the First 65,000 Infants

(2018) Melissa P. Wasserstein et al.   GENETICS IN MEDICINE

Effects of Enzyme Replacement Therapy and Antidrug Antibodies in Patients with Fabry Disease

(2018) Malte Lenders et al.   JOURNAL OF THE AMERICAN SOCIETY OF NEPHROLOGY

Biopharmaceutical benchmarks 2018

(2018) Gary Walsh   NATURE BIOTECHNOLOGY

Lysosomal storage diseases

(2018) Frances M. Platt et al.   Nature Reviews Disease Primers

Newborn screening for lysosomal storage disorders by tandem mass spectrometry in North East Italy

(2017) Alberto B. Burlina et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Emptying the stores: lysosomal diseases and therapeutic strategies

(2017) Frances M. Platt   NATURE REVIEWS DRUG DISCOVERY

Achieving the Promise of Therapeutic Extracellular Vesicles: The Devil is in Details of Therapeutic Loading

(2017) Dhruvitkumar S. Sutaria et al.   PHARMACEUTICAL RESEARCH

Bacterial Inclusion Bodies: Discovering Their Better Half

(2017) Ursula Rinas et al.   TRENDS IN BIOCHEMICAL SCIENCES

Highly Versatile Polyelectrolyte Complexes for Improving the Enzyme Replacement Therapy of Lysosomal Storage Disorders

(2016) Marina I. Giannotti et al.   ACS Applied Materials & Interfaces

Oral pharmacological chaperone migalastat compared with enzyme replacement therapy in Fabry disease: 18-month results from the randomised phase III ATTRACT study

(2016) Derralynn A Hughes et al.   JOURNAL OF MEDICAL GENETICS

α-Galactosidase-A Loaded-Nanoliposomes with Enhanced Enzymatic Activity and Intracellular Penetration

(2016) Ingrid Cabrera et al.   Advanced Healthcare Materials

Strategies for the production of difficult-to-express full-length eukaryotic proteins using microbial cell factories: production of human alpha-galactosidase A

(2015) Ugutz Unzueta et al.   APPLIED MICROBIOLOGY AND BIOTECHNOLOGY

A novel bio-functional material based on mammalian cell aggresomes

(2015) Escarlata Rodríguez-Carmona et al.   APPLIED MICROBIOLOGY AND BIOTECHNOLOGY

Mannose receptor-mediated delivery of moss-made α-galactosidase A efficiently corrects enzyme deficiency in Fabry mice

(2015) Jin-Song Shen et al.   JOURNAL OF INHERITED METABOLIC DISEASE

Lysosomal Storage Disorder Screening Implementation: Findings from the First Six Months of Full Population Pilot Testing in Missouri

(2015) Patrick V. Hopkins et al.   JOURNAL OF PEDIATRICS

Characterization of a chemically modified plant cell culture expressed human α-Galactosidase-A enzyme for treatment of Fabry disease

(2015) Tali Kizhner et al.   MOLECULAR GENETICS AND METABOLISM

Polymer Capsules as a Theranostic Tool for a Universal In Vitro Screening Assay-The Case of Lysosomal Storage Diseases

(2015) Moritz Nazarenus et al.   PARTICLE & PARTICLE SYSTEMS CHARACTERIZATION

Carboxyl-Terminal Truncations Alter the Activity of the Human α-Galactosidase A

(2015) Mariam Meghdari et al.   PLoS One

The effectiveness and cost-effectiveness of enzyme and substrate replacement therapies: a longitudinal cohort study of people with lysosomal storage disorders.

(2014) K Wyatt et al.   HEALTH TECHNOLOGY ASSESSMENT

Enzyme replacement therapy attenuates disease progression in a canine model of late-infantile neuronal ceroid lipofuscinosis (CLN2 disease)

(2014) Martin L. Katz et al.   JOURNAL OF NEUROSCIENCE RESEARCH

Enhancing Biodistribution of Therapeutic Enzymes In Vivo by Modulating Surface Coating and Concentration of ICAM-1-Targeted Nanocarriers

(2013) Janet Hsu et al.   Journal of Biomedical Nanotechnology

Extracellular vesicles: Exosomes, microvesicles, and friends

(2013) Graça Raposo et al.   JOURNAL OF CELL BIOLOGY

Multifunctional Nanovesicle-Bioactive Conjugates Prepared by a One-Step Scalable Method Using CO2-Expanded Solvents

(2013) Ingrid Cabrera et al.   NANO LETTERS

Specific Transfection of Inflamed Brain by Macrophages: A New Therapeutic Strategy for Neurodegenerative Diseases

(2013) Matthew J. Haney et al.   PLoS One

Unconventional microbial systems for the cost-efficient production of high-quality protein therapeutics

(2012) José Luis Corchero et al.   BIOTECHNOLOGY ADVANCES

CNS Penetration of Intrathecal-Lumbar Idursulfase in the Monkey, Dog and Mouse: Implications for Neurological Outcomes of Lysosomal Storage Disorder

(2012) Pericles Calias et al.   PLoS One

Long-Term Effect of Antibodies against Infused Alpha-Galactosidase A in Fabry Disease on Plasma and Urinary (lyso)Gb3 Reduction and Treatment Outcome

(2012) Saskia M. Rombach et al.   PLoS One

Recent advances in stealth coating of nanoparticle drug delivery systems

(2012) Zohreh Amoozgar et al.   Wiley Interdisciplinary Reviews-Nanomedicine and Nanobiotechnology

pH-Responsive Polysaccharide-Based Polyelectrolyte Complexes As Nanocarriers for Lysosomal Delivery of Therapeutic Proteins

(2011) Marina I. Giannotti et al.   BIOMACROMOLECULES

Integrated approach to produce a recombinant, his-tagged human α-galactosidase a in mammalian cells

(2011) José Luis Corchero et al.   BIOTECHNOLOGY PROGRESS

The impact of antibodies on clinical outcomes in diseases treated with therapeutic protein: Lessons learned from infantile Pompe disease

(2011) Suhrad G. Banugaria et al.   GENETICS IN MEDICINE

Production of active human glucocerebrosidase in seeds of Arabidopsis thaliana complex-glycan-deficient (cgl) plants

(2011) Xu He et al.   GLYCOBIOLOGY

Biological activities of histidine-rich peptides; merging biotechnology and nanomedicine

(2011) Neus Ferrer-Miralles et al.   Microbial Cell Factories

Glycosylation of Therapeutic Proteins

(2010) Ricardo J. Solá et al.   BIODRUGS

Enhanced endothelial delivery and biochemical effects of α-galactosidase by ICAM-1-targeted nanocarriers for Fabry disease

(2010) Janet Hsu et al.   JOURNAL OF CONTROLLED RELEASE

Delivery of Therapeutic Proteins

(2010) Dipak S. Pisal et al.   JOURNAL OF PHARMACEUTICAL SCIENCES

Fabry disease

(2010) Dominique P Germain   Orphanet Journal of Rare Diseases

New biotechnological and nanomedicine strategies for treatment of lysosomal storage disorders

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

Glycan Engineering and Production of ‘Humanized’ Glycoprotein in Yeast Cells

(2009) Yasunori Chiba et al.   BIOLOGICAL & PHARMACEUTICAL BULLETIN

Vasculopathy in patients with Fabry disease: Current controversies and research directions

(2009) S.M. Rombach et al.   MOLECULAR GENETICS AND METABOLISM

Globotriaosylceramide induces oxidative stress and up-regulates cell adhesion molecule expression in Fabry disease endothelial cells

(2008) Jin-Song Shen et al.   MOLECULAR GENETICS AND METABOLISM