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Publications by Karlaina J.L. Osmon
711. Intravenous Neonatal Gene Therapy Corrects GM2 Gangliosidosis in Sandhoff Mice for Long-Term, by Using AAV Viral Vector Expressing a New Hexosaminidase Variant
Molecular Therapy
Molecular Medicine
Molecular Biology
Pharmacology
Medicine
Genetics
Drug Discovery
Related publications
Deficiency of the Hexosaminidase a Activator Protein in a Case of GM2 Gangliosidosis; Variant AB
Pediatric Research
Child Health
Pediatrics
Perinatology
Partial Deficiency of Beta-Hexosaminidase Activity in Canine GM2-gangliosidosis.
Tohoku Journal of Experimental Medicine
Biochemistry
Medicine
Genetics
Molecular Biology
Biochemical Characterization of the GM2 Gangliosidosis B1 Variant
Brazilian Journal of Medical and Biological Research
Immunology
Cell Biology
Pharmacology
Biochemistry
Biophysics
Neuroscience
Medicine
Toxicology
Physiology
Pharmaceutics
GM2-Gangliosidosis, AB Variant: Clinical, Ophthalmological, MRI, and Molecular Findings
JIMD Reports
Internal Medicine
Genetics
Molecular Biology
Biochemistry
Endocrinology
Metabolism
Diabetes
CFTR Gene Transduction in Neonatal Rabbits Using Anadeno-Associated Virus (AAV) Vector
Gene Therapy
Molecular Medicine
Genetics
Molecular Biology
159. Long-Term Rescue by AAV-Delivered Therapy in Autosomal Dominant Retinitis Pigmentosa (ADRP) Mice
Molecular Therapy
Molecular Medicine
Molecular Biology
Pharmacology
Medicine
Genetics
Drug Discovery
AAV Gene Therapy Corrects OTC Deficiency and Prevents Liver Fibrosis in Aged OTC-knock Out Heterozygous Mice
Molecular Genetics and Metabolism
Genetics
Molecular Biology
Biochemistry
Endocrinology
Metabolism
Diabetes
377. Significant Therapeutic Benefit After Post-Symptomatic Gene Therapy in a Feline Model of GM2 Gangliosidosis
Molecular Therapy
Molecular Medicine
Molecular Biology
Pharmacology
Medicine
Genetics
Drug Discovery
Retrospective Diagnosis of Feline GM2 Gangliosidosis Variant 0 (Sandhoff-Like Disease) in Japan: Possible Spread of the Mutant Allele in the Japanese Domestic Cat Population
Journal of Veterinary Medical Science
Veterinary