Preclinical dose-finding study with a liver-tropic, recombinant AAV-2/8 vector in the mouse model of galactosialidosis

Medicine and Dentistry

• Protein 83%
• Liver 50%
• Therapeutic Procedure 33%
• Systemic Disease 33%
• Patient 33%
• Infertility 33%
• Organ 33%
• Diseases 16%
• Serum 16%
• Age 16%
• Phenotype 16%
• Urine 16%
• Promoter Region 16%
• Lifespan 16%
• Gene Targeting 16%
• Transgene 16%
• Nephropathy 16%
• Nervous System 16%
• Ataxia 16%
• Reticuloendothelial System 16%
• Lysosomal Storage Disease 16%
• Guanosine Phosphate 16%
• Reproductive Organ 16%
• Symptom 16%
• Side Effect 16%

Biochemistry, Genetics and Molecular Biology

• Galactosialidosis 100%
• Protein 83%
• Dose 66%
• Cathepsin A 66%
• Mouse 50%
• Mouse Model 16%
• Age 16%
• Phenotype 16%
• Promoter Region 16%
• Knockout Mouse 16%
• Lifespan 16%
• Gene Targeting 16%
• Lysosomal Storage Disease 16%
• Physical Appearance 16%

Pharmacology, Toxicology and Pharmaceutical Science

• Protein 83%
• Mouse 50%
• Infertility 33%
• Systemic Disease 33%
• Diseases 16%
• Mouse Model 16%
• Symptom 16%
• Knockout Mouse 16%
• Guanosine Phosphate 16%
• Kidney Disease 16%
• Gene Delivery 16%
• Ataxia 16%
• Lysosome Storage Disease 16%
• Side Effect 16%