Compositions and methods for diagnosing and treating peroxisomal diseases
US-2020278356-A1 · Sep 3, 2020 · US
Compositions and methods for treating lysosomal storage diseases and disorders
US11548936B2 · US · B2
| Field | Value |
|---|---|
| Publication number | US-11548936-B2 |
| Application number | US-201816477829-A |
| Country | US |
| Kind code | B2 |
| Filing date | Jan 16, 2018 |
| Priority date | Jan 17, 2017 |
| Publication date | Jan 10, 2023 |
| Grant date | Jan 10, 2023 |
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- Title
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Abstract
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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.
First claim
Opening claim text (preview).
What is claimed is: 1. 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. 2. The method of claim 1 , wherein the subject is pre-selected by detecting an increase in the level of a metallothionein (MT) polynucleotide or polypeptide in a sample of the subject relative to a reference. 3. The method of claim 1 , wherein the metallothionein is selected from the group consisting of metallothionein-1A (MT1A), metallothionein-1B (MT1B), metallothionein-1E (MT1E), metallothionein-1F (MT1F), metallothionein-1G (MT1G), metallothionein-1H (MT1H), metallothionein-lI pseudogene (MT1Ip or MTE), metallothionein-1L (LT1L or MT1R), metallothionem-1M (MT1M or MT1K), and metallothionein-1X (MT1X). 4. The method of claim 1 , wherein the method comprises administering the AAV vector to the subject. 5. The method of claim 4 , wherein the AAV vector is administered systemically. 6. The method of claim 1 , wherein the method comprises administering the LV vector. 7. The method of claim 6 , wherein the LV vector is administered systemically. 8. 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. 9. The method of claim 8 , wherein the subject is pre-selected by detecting an increase in the level of a metallothionein (MT) polynucleotide or polypeptide in a sample of the subject relative to a reference. 10. The method of claim 8 , wherein the metallothionein selected from the group consisting of metallothionein-1A (MT1A), metallothionein-1B (MT1B), metallothionein-1E (MT1E), metallothionein-1F (MT1F), metallothionein-1G (MT1G), metallothionein-1H (MT1H), metallothionein-lI pseudogene (MT1Ip or MTE), metallothionein-1L (LT1L or MT1R), metallothionem-1M (MT1M or MT1K), and metallothionein-1X (MT1X). 11. The method of claim 8 , wherein the vector is a lentiviral vector or an adeno associated virus (AAV) vector.
Assignees
Inventors
Classifications
- C07K14/825Primary
Metallothioneins · CPC title
- A61P43/00Primary
Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00 · CPC title
viral genome or elements thereof as genetic vector · CPC title
Endocrine or metabolic disorders · CPC title
viral genome or elements thereof as genetic vector · CPC title
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- What does patent US11548936B2 cover?
- 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.
- Who is the assignee on this patent?
- Childrens Medical Center, Ospedale San Raffaele Srl, Dana Farber Cancer Inst Inc, and 1 more
- What technology area does this patent fall under?
- Primary CPC classification C07K14/825. Mapped technology areas include Chemistry & Metallurgy.
- When was this patent published?
- Publication date Tue Jan 10 2023 00:00:00 GMT+0000 (Coordinated Universal Time) (B2). Legal status and post-grant events are not shown on this page.
- What related patents are in patentsdb?
- We list 5 related publications on this page (citations in our corpus or others sharing the same primary CPC).