In vivo selection in non-human primates identifies AAV capsids for on-target CSF delivery to spinal cordMOLECULAR THERAPYIF122024-08-011OAAI
Comparative Effectiveness of Intracerebroventricular, Intrathecal, and Intranasal Routes of AAV9 Vector Administration for Genetic Therapy of Neurologic Disease in Murine Mucopolysaccharidosis Type IFRONTIERS IN MOLECULAR NEUROSCIENCEIF3.82021-05-1034OAAI
AAV8-antiVEGFfab Ocular Gene Transfer for Neovascular Age-Related Macular DegenerationMOLECULAR THERAPYIF122018-02-0147OAAI
Neurologic improvement in a mouse model of Hunter syndrome (mucopolysaccharidosis type II) by treatment with AAV9 vector after the development of cognitive dysfunctionMOLECULAR GENETICS AND METABOLISMIF3.52017-01-010PREAI
Relative effectiveness of different routes of AAV administration for gene therapy of mucopolysaccharidosisMOLECULAR GENETICS AND METABOLISMIF3.52017-01-010PREAI
AAV9 Delivery into Cerebrospinal Fluid Corrects CNS Disease in a Murine Model of Mucopolysaccharidosis Type IIMOLECULAR THERAPYIF122016-05-010OAAI
Intranasal Gene Delivery of AAV Iduronidase: An Effective and Non-Invasive Approach for Treatmentof CNS Disease in a Murine Model of MPS Type IMOLECULAR THERAPYIF122016-05-010OAAI
Therapeutic Effectiveness of AAV-Mediated Iduronidase Delivery to the CNS Following Intravenous Administration in a Murine Model of Mucopolysaccharidosis Type IMOLECULAR THERAPYIF122016-05-010OAAI
GLP-Compliant Non-Clinical Safety and Biodistribution of a Recombinant AAV2/8 Vector Administered Intravenously for Treatment of Mucopolysaccharidosis Type VIMOLECULAR THERAPYIF122016-05-012OAAI
AAV9 Mediated Correction of Iduronate-2-Sulfatase Deficiency in the Central Nervous System to Prevent the Onset of Neurologic Deficits in a Murine Model of Mucopolysaccharidosis Type II.MOLECULAR THERAPYIF122016-05-010OAAI
Gene delivery and biodistribution following intravenous administration of AAV9/rh10 iduronidase in a murine model of MPS type IMOLECULAR GENETICS AND METABOLISMIF3.52016-02-010PREAI
Intranasal gene delivery of AAV9 iduronidase: A non-invasive and effective gene therapy approach for prevention of neurologic disease in a murine model of mucopolysaccharidosis type IMOLECULAR GENETICS AND METABOLISMIF3.52016-02-010PREAI
AAV9 mediated correction of iduronate-2-sulfatase deficiency in the central nervous system of mucopolysaccharidosis type II miceMOLECULAR GENETICS AND METABOLISMIF3.52016-02-010PREAI
AAV9 Mediated Correction of Iduronate-2-Sulfatase Deficiency in the Central Nervous System of Mucopolysaccharidosis Type II MiceMOLECULAR THERAPYIF122015-05-010OAAI
Non-Invasive Intranasal Administration of AAV9-Iduronidase Prevents Emergence of Neurologic Disease and Neurocognitive Dysfunction in a Murine Model of Mucopolysaccharidosis Type IMOLECULAR THERAPYIF122015-05-010OAAI
Prevalence of Anti-AAV8 Neutralizing Antibodies and ARSB Cross-Reactive Immunologic Material in MPS VI Patients Candidates for a Gene Therapy TrialMOLECULAR THERAPYIF122015-05-010OAAI
Intrathecal and intranasal infusion of adeno-associated virus vector: non-invasive routes of administration achieving corrective levels of iduronidase expression throughout the brain for gene therapy of mucopolysaccharidosis type IMOLECULAR GENETICS AND METABOLISMIF3.52014-02-010PREAI
The lipidation status of acute-phase protein serum amyloid A determines cholesterol mobilization via scavenger receptor class B, type IBIOCHEMICAL JOURNALIF4.32007-01-2549OAAI
Sterol carrier protein 2 gene transfer changes lipid metabolism and enterohepatic sterol circulation in miceGASTROENTEROLOGYIF25.12000-12-0134OA
Scavenger receptor class B, type I-mediated [3H]cholesterol efflux to high and low density lipoproteins is dependent on lipoprotein binding to the receptorJOURNAL OF BIOLOGICAL CHEMISTRYIF3.92000-09-01185OAAI
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