X
Search Filters
Format Format
Subjects Subjects
Subjects Subjects
X
Sort by Item Count (A-Z)
Filter by Count
humans (9) 9
index medicus (8) 8
metachromatic leukodystrophy (7) 7
mice (6) 6
animals (5) 5
genetics & heredity (5) 5
cerebroside-sulfatase - genetics (4) 4
leukodystrophy, metachromatic - diagnosis (4) 4
mouse model (4) 4
arylsulfatase a (3) 3
expression (3) 3
gene-therapy (3) 3
genetic therapy - methods (3) 3
genotype (3) 3
hematopoietic stem cell transplantation (3) 3
hematopoietic stem cells - metabolism (3) 3
leukodystrophy, metachromatic - genetics (3) 3
mice, inbred c57bl (3) 3
transplantation (3) 3
abridged index medicus (2) 2
alleles (2) 2
apoptosis (2) 2
arsa (2) 2
biomarkers - metabolism (2) 2
central-nervous-system (2) 2
chronic granulomatous-disease (2) 2
clinical neurology (2) 2
coculture techniques (2) 2
disease (2) 2
disease models, animal (2) 2
enzymes (2) 2
gene therapy (2) 2
hematology (2) 2
hematopoietic stem cells (2) 2
leukocytes, mononuclear - metabolism (2) 2
leukodystrophy, metachromatic - metabolism (2) 2
lysosomal storage diseases (2) 2
lysosomal storage diseases - diagnosis (2) 2
lysosomal storage diseases - metabolism (2) 2
lysosomal storage diseases - pathology (2) 2
metallothionein - chemistry (2) 2
mice, knockout (2) 2
microglia (2) 2
missense mutations (2) 2
molecular dynamics simulation (2) 2
mutation (2) 2
nervous system diseases (2) 2
neurosciences (2) 2
pharmacology (2) 2
phenotype (2) 2
primary cell culture (2) 2
proteins (2) 2
rodents (2) 2
severe combined immunodeficiency (2) 2
sphingolipidoses (2) 2
a pseudodeficiency (1) 1
activator protein (1) 1
adrenoleukodystrophy - genetics (1) 1
adrenoleukodystrophy - therapy (1) 1
analysis (1) 1
animal models (1) 1
apoptosis - drug effects (1) 1
arylsulfatase-a gene (1) 1
arylsulfatase-a pseudodeficiency (1) 1
arylsulphatase-b (1) 1
assay (1) 1
biochemistry & molecular biology (1) 1
blood (1) 1
blood cd34 cells (1) 1
bone marrow - enzymology (1) 1
bone marrow - metabolism (1) 1
bone-marrow (1) 1
bone-marrow-transplantation (1) 1
brain - pathology (1) 1
brain disease (1) 1
cd4 t-cells (1) 1
cdna integration (1) 1
cell gene-therapy (1) 1
cell survival - drug effects (1) 1
cells (1) 1
cells, cultured (1) 1
central nervous system (1) 1
cerebroside-sulfatase - blood (1) 1
chemical compounds (1) 1
chemistry (1) 1
clinical trials as topic (1) 1
databases, genetic (1) 1
degeneration (1) 1
demyelinating diseases (1) 1
diagnosis (1) 1
disorders (1) 1
dna damage (1) 1
dna-binding proteins - deficiency (1) 1
dna-binding proteins - genetics (1) 1
drug discovery (1) 1
enzymatic activity (1) 1
ex vivo manipulation (1) 1
ex-vivo expansion (1) 1
female (1) 1
flow cytometry (1) 1
more...
Language Language
Publication Date Publication Date
Click on a bar to filter by decade
Slide to change publication date range


Science, ISSN 0036-8075, 8/2013, Volume 341, Issue 6148, pp. 864 - 864
Journal Article
Journal Article
Human Mutation, ISSN 1059-7794, 01/2016, Volume 37, Issue 1, pp. 16 - 27
Journal Article
Annals of Neurology, ISSN 0364-5134, 01/2014, Volume 75, Issue 1, pp. 127 - 137
Objective: To facilitate development of novel disease-modifying therapies for lysosomal storage disorder (LSDs) characterized by nervous system involvement... 
TWITCHER MOUSE | METACHROMATIC LEUKODYSTROPHY | STORAGE DISORDERS | CENTRAL-NERVOUS-SYSTEM | GENE-THERAPY | MODEL | SANDHOFF-DISEASE | EXPRESSION | NEUROSCIENCES | CLINICAL NEUROLOGY | PARKINSONS-DISEASE | BLOOD | Disease | Patients | Rodents | Original
Journal Article
Journal Article
Molecular Therapy, ISSN 1525-0016, 2006, Volume 13, Issue S1, pp. S157 - S157
  In the past years we worked extensively on the development of HSC gene therapy for two fatal demyelinating lysosomal storage disorders, Metachromatic (MLD)... 
Journal Article
03/2013
A method of diagnosis or prognosis of a lysosomal storage disorder in a subject, wherein said method comprises (i) providing a sample from said subject, (ii)... 
TESTING | METALLURGY | PEPTIDES | INVESTIGATING OR ANALYSING MATERIALS BY DETERMINING THEIRCHEMICAL OR PHYSICAL PROPERTIES | ORGANIC CHEMISTRY | CHEMISTRY | MEASURING | PHYSICS
Patent
No results were found for your search.

Cannot display more than 1000 results, please narrow the terms of your search.