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CNS-directed gene therapy for lysosomal storage diseases
Mark S. Sands
, Mark E. Haskins
Center of Regenerative Medicine
Roy and Diana Vagelos Division of Biology & Biomedical Sciences (DBBS)
Institute of Clinical and Translational Sciences (ICTS)
Siteman Cancer Center
Bursky Center for Human Immunology & Immunotherapy Programs (CHiiPs)
DBBS - Molecular Cell Biology
DBBS - Neurosciences
DBBS - Molecular Genetics and Genomics
Hope Center for Neurological Disorders
Intellectual and Developmental Disabilities Research Center (IDDRC)
Division of Oncology
Research output
:
Contribution to journal
›
Article
›
peer-review
59
Scopus citations
Overview
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Keyphrases
Anterograde Axonal Transport
12%
Bone Marrow Transplantation
25%
Canine
12%
Central Nervous System
100%
Disease-associated
12%
Distant Site
12%
Enzyme Replacement Therapy
12%
Feline
12%
Functional Improvement
12%
Gene Therapy
100%
Gene Transfer Vector
25%
Inherited Metabolic Diseases
12%
Intracranial Injection
12%
Lysosomal Enzyme Trafficking
12%
Lysosomal Enzymes
37%
Lysosomal Storage Disease
100%
Multi-enzyme
12%
Multiple Organs
12%
Nervous System Diseases
37%
Protein Transduction Domain
25%
Recombinant Enzyme
12%
Retrograde Axonal Transport
12%
Small Rodents
12%
Storage Innovations
12%
System Components
25%
Systemic Disease
12%
Therapeutic Levels
12%
Treatment Efficacy
12%
Viral-mediated Gene Transfer
12%
Neuroscience
Axoplasmic Transport
20%
Central Nervous System
100%
Central Nervous System Disease
60%
Enzyme Replacement Therapy
20%
In Vivo
20%
Lysosome Enzyme
80%
Metabolic Disorder
20%
Recombinant Enzyme
20%
Retrograde Nerve Fiber Transport
20%
Shuttle Vector
40%
Viral Gene Delivery System
20%
Biochemistry, Genetics and Molecular Biology
Animal Model
11%
Cat
11%
Enzyme
100%
Gene Therapy
100%
Hope
11%
Lysosomal Storage Disease
100%
Metabolic Disorder
11%
Nerve Fiber Transport
11%
Retrograde Nerve Fiber Transport
11%
Shuttle Vector
22%
Viral Gene Delivery System
11%
Pharmacology, Toxicology and Pharmaceutical Science
Cat
12%
Central Nervous System Disease
37%
Combination Therapy
12%
Lysosome Enzyme
50%
Lysosome Storage Disease
100%
Metabolic Disorder
12%
Recombinant Enzyme
12%
Replacement Therapy
12%
Systemic Disease
12%