Keyphrases
Mucopolysaccharidosis VII
100%
Lysosomal Storage Disease
99%
Murine Model
70%
Gene Therapy
66%
Neuronal Ceroid Lipofuscinosis
54%
β-glucuronidase
52%
Bone Marrow Transplantation
37%
Enzyme Replacement Therapy
36%
Krabbe Disease
34%
Globoid Cell Leukodystrophy
33%
Acyl Protein Thioesterase
29%
Liver
27%
Central Nervous System
24%
Psychosine
21%
Mouse Model
20%
AAV2
20%
Lysosomal Enzymes
20%
Gene Transfer
18%
Spleen
16%
PPT1
16%
Adenovirus
15%
Spinal Cord
14%
CLN1 Disease
13%
Galactosylceramidase
13%
Twitcher Mouse
12%
Retroviral Vector
11%
Galactocerebrosidase
11%
Photosystem II
11%
Therapeutic Efficacy
11%
Glycosaminoglycans
11%
Hematopoietic Stem Cells
10%
Macrophages
10%
Xenotransplantation Model
10%
Therapeutic Levels
10%
TFIIIA
9%
Recombinant Adeno-associated Virus (rAAV)
9%
Lentiviral
8%
Recombinant
8%
Sanfilippo Syndrome
8%
Motor Function
8%
Neurodegenerative Diseases
8%
Gene-deficient Mice
8%
Clinical Course
7%
Syngeneic
7%
Lentiviral Vector
7%
Adeno-associated Viral Vector
7%
Pathogenic Mechanism
7%
MPS VII
7%
Juvenile Batten Disease
7%
High-level Expression
7%
Biochemistry, Genetics and Molecular Biology
Mouse
72%
Sly Syndrome
53%
Enzyme
47%
Gene Therapy
45%
Lysosomal Storage Disease
36%
Beta-Glucuronidase
32%
Krabbe Disease
23%
Galactosylceramidase
18%
Lifespan
16%
Infantile Neuronal Ceroid Lipofuscinosis
15%
Palmitoyl Protein Thioesterase
14%
GUSB
13%
Batten Disease
13%
Neuronal Ceroid Lipofuscinosis
12%
Retrovirus Vector
11%
Transfer Gene
10%
Adeno Associated Virus Vector
10%
Hematopoietic Stem Cell
9%
Macrophage
9%
Stem Cell
8%
Glycosaminoglycan
8%
Twitcher Mouse
8%
5S Ribosomal RNA
7%
Enzyme Activity
6%
Adeno Associated Virus
6%
Mouse Model
6%
Lysosome
6%
Hepatocyte
6%
Promoter Region
5%
Hematopoietic Stem Cell Transplantation
5%
Animal Model
5%
Autosomal Dominant Inheritance
5%
Astrocyte
5%
Transgene
5%
Medicine and Dentistry
Sly Syndrome
61%
Lysosomal Storage Disease
51%
Gene Therapy
40%
Disease
36%
Beta Glucuronidase
31%
Infantile Neuronal Ceroid Lipofuscinosis
26%
Enzyme Replacement Therapy
24%
Bone Marrow Transplantation
19%
Palmitoyl Protein Thioesterase
16%
Adeno-Associated Virus
13%
Neonatal Infant
11%
Lifespan
11%
Globoid Cell Leukodystrophy
10%
Central Nervous System
10%
Lysosome Enzyme
9%
Gene Transfer
9%
Disease Course
9%
Macrophage
9%
Mucopolysaccharidosis
8%
Childbirth
8%
Combination Therapy
8%
Hematopoietic Stem Cell
8%
Retrovirus Vector
8%
Batten Disease
7%
Adeno Associated Virus Vector
7%
Epileptic Seizure
7%
Intravenous Drug Administration
6%
Sanfilippo Syndrome
5%
Hepatocyte
5%
