- AAV9
- Agonist
- Autophagy
- Biomarker
- Clinical Trial
- CNS
- CoRDS
- CRISPR
- Dendrites
- Electromyography (EMG)
- Endpoint
- FMRI
- Gene therapy
- Genotype
- Hematopoietic stem cells
- Hypomylenation
- Institutional Review Board (IRB)
- Investigational New Drug Application (IND)
- Leukodystophy
- Lumbar Puncture
- Lysosomal storage disease
- Lysosome
- Magnetic Resonance Imaging (MRI)
- Microglia
- Mucolipin-1 or MCOLN1
- Mutation
- Natural History Study
- Neurodegenerative Disease
- Neuroinflammation
- Oligodendrocytes
- Optical Coherence Tomography (OCT)
- Pathogenesis
- Pathway
- Phenotype
- PI
- Prospective Natural History Study
- Purkinjie cell
- Registry
- Retrospective Natural History Study
- Small molecule
- Stem cells
- Substrate reduction therapy
- Target
- TFEB
- Transduction
- Transfection
- Translational
- Transmembrane protein defect
- TRPML1
- Tx
- Vector
- Wild type
