New biotechnological and nanomedicine strategies for treatment of lysosomal storage disorders

Gene Therapy for Lysosomal Storage Diseases (LSDs) in Large Animal Models

(2013) M. Haskins   ILAR JOURNAL

Sphingomyelinases: their regulation and roles in cardiovascular pathophysiology

(2009) Catherine Pavoine et al.   CARDIOVASCULAR RESEARCH

CNS-directed gene therapy for lysosomal storage diseases

(2008) Mark S Sands et al.   ACTA PAEDIATRICA

RhoA activation and actin reorganization involved in endothelial CAM-mediated endocytosis of anti-PECAM carriers: critical role for tyrosine 686 in the cytoplasmic tail of PECAM-1

(2008) C. Garnacho et al.   BLOOD

Oxygen Microscopy by Two-Photon-Excited Phosphorescence

(2008) Olga S. Finikova et al.   CHEMPHYSCHEM

Differential intra-endothelial delivery of polymer nanocarriers targeted to distinct PECAM-1 epitopes

(2008) C GARNACHO et al.   JOURNAL OF CONTROLLED RELEASE

Non-inhibitory antibodies impede lysosomal storage reduction during enzyme replacement therapy of a lysosomal storage disease

(2008) Ulrich Matzner et al.   JOURNAL OF MOLECULAR MEDICINE-JMM

Delivery of Acid Sphingomyelinase in Normal and Niemann-Pick Disease Mice Using Intercellular Adhesion Molecule-1-Targeted Polymer Nanocarriers

(2008) C. Garnacho et al.   JOURNAL OF PHARMACOLOGY AND EXPERIMENTAL THERAPEUTICS

Reduced α-Gal A enzyme activity in Fabry fibroblast cells and Fabry mice tissues induced by serum from antibody positive patients with Fabry disease

(2008) Toya Ohashi et al.   MOLECULAR GENETICS AND METABOLISM

Mannose 6-Phosphate Receptor–mediated Transport of Sulfamidase Across the Blood–brain Barrier in the Newborn Mouse

(2008) Akihiko Urayama et al.   MOLECULAR THERAPY

Control of Endothelial Targeting and Intracellular Delivery of Therapeutic Enzymes by Modulating the Size and Shape of ICAM-1-targeted Carriers

(2008) Silvia Muro et al.   MOLECULAR THERAPY

Targeting of the CNS in MPS-IH Using a Nonviral Transferrin-α-l-iduronidase Fusion Gene Product

(2008) Mark J Osborn et al.   MOLECULAR THERAPY

Cellular uptake and lysosomal delivery of galactocerebrosidase tagged with the HIV Tat protein transduction domain

(2007) Xian-Yang Zhang et al.   JOURNAL OF NEUROCHEMISTRY