Compositions and methods for treating lysosomal storage diseases and disorders
AssigneesChildren's Medical Center Corporation, Ospedale San Raffaele S.R.L., Dana-Farber Cancer Institute, Inc., Fondazione Telethon
InventorsAlessandra Biffi, Eleonora Cavalca
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Abstract
The present invention provides compositions and methods for the treatment or prevention of a lysosomal disease or disorder involving increasing the level, expression, or activity of a metallothionein polypeptide or polynucleotide in the subject.
Claims11
A method of treating a lysosomal storage disease or disorder in a subject, the method comprising increasing the level, expression, or activity of a metallothionein-1 polypeptide or polynucleotide in the subject relative to a reference by administering to the subject an adeno-associated virus (AAV) vector or a lentiviral (LV) vector encoding the metallothionein-1 polypeptide or polynucleotide, wherein the lysosomal storage disease is globoid leukodystrophy or metachromatic leukodystrophy.
A method of treating a lysosomal storage disease or disorder in a subject, the method comprising increasing the level, expression, or activity of a metallothionein-1 polypeptide or polynucleotide in the subject relative to a reference by administering to the subject hematopoietic stem cells (HSCs) comprising a vector encoding the metallothionein-1 polypeptide or polynucleotide, wherein the lysosomal storage disease is globoid leukodystrophy or metachromatic leukodystrophy.
Dependencies are inferred from the claim text.