A novel mouse model of a patient mucolipidosis II mutation recapitulates disease pathology. [electronic resource]
Producer: 20141211Description: 26709-26721 p. digitalISSN:- 1083-351X
- 2-Hydroxypropyl-beta-cyclodextrin
- Animals
- Behavior, Animal
- Carrier Proteins -- genetics
- Disease Models, Animal
- Excipients -- pharmacology
- Glycoproteins -- genetics
- HEK293 Cells
- Homozygote
- Humans
- Mice
- Mice, Inbred BALB C
- Mice, Mutant Strains
- Mucolipidoses -- enzymology
- Mutation
- Niemann-Pick Disease, Type C -- enzymology
- Purkinje Cells -- enzymology
- Transferases (Other Substituted Phosphate Groups) -- genetics
- Vesicular Transport Proteins -- genetics
- beta-Cyclodextrins -- pharmacology
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Publication Type: Journal Article; Research Support, Non-U.S. Gov't
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