Keyphrases
Gene Therapy
100%
Whole-body
100%
Mucopolysaccharidosis Type IIIA
100%
Sulfamidase
80%
Lysosomal Storage Disease
60%
AAV9
60%
Glycosaminoglycans
40%
Enzymatic Activity
20%
Large Mammals
20%
Short Half-life
20%
Gene Transfer
20%
Adeno-associated Viral Vector
20%
Heparan Sulfate
20%
Liver
20%
Effective Therapy
20%
Beagle Dog
20%
Antibody Titer
20%
CNS Involvement
20%
Transgenic Expression
20%
Clinical Translation
20%
Sulfatase
20%
Behavioral Deficits
20%
Serotype 9
20%
High Titer
20%
Serum Antibody
20%
Lysosomal Enzymes
20%
Prolonged Survival
20%
Somatic Pathology
20%
Affected children
20%
Fluid Administration
20%
CNS Pathologies
20%
Drug Bioavailability
20%
AAV Antibody
20%
Intracerebroventricular Injection
20%
Intracisternal
20%
Stepwise Degradation
20%
Biochemistry, Genetics and Molecular Biology
Gene Therapy
100%
Liquid
100%
Mucopolysaccharidosis
100%
Lysosomal Storage Disease
60%
Titer
40%
Glycosaminoglycan
40%
Enzyme
20%
Adeno Associated Virus Vector
20%
Transgenics
20%
Enzyme Activity
20%
Blood Brain Barrier
20%
Serotype
20%
Sulfatase
20%
Antibody Blood Level
20%
Beagle
20%
Heparan Sulfate
20%
Pharmacology, Toxicology and Pharmaceutical Science
Mucopolysaccharidosis
100%
N Sulfoglucosamine Sulfohydrolase
80%
Lysosome Storage Disease
60%
Glycosaminoglycan
40%
Beagle
20%
Bioavailability
20%
Adeno Associated Virus Vector
20%
Gene Delivery
20%
Sulfatase
20%
Heparan Sulfate
20%
Lysosome Enzyme
20%
Blood-Brain Barrier
20%
Neuroscience
N Sulfoglucosamine Sulfohydrolase
100%
Glycosaminoglycan
50%
Gene Delivery
25%
Somatics
25%
Heparan Sulfate
25%
Gene Transfer
25%
Blood Brain Barrier
25%
Enzyme Activity
25%
Adeno Associated Virus Vector
25%
Adeno-Associated Virus
25%
Lysosome Enzyme
25%
Sulfatase
25%