Gene therapy for neurometabolic disorders
US-2017152524-A1 · Jun 1, 2017 · US
Gene therapy for lysosomal storage diseases
US11369693B2 · US · B2
| Field | Value |
|---|---|
| Publication number | US-11369693-B2 |
| Application number | US-201414328621-A |
| Country | US |
| Kind code | B2 |
| Filing date | Jul 10, 2014 |
| Priority date | Jun 6, 2007 |
| Publication date | Jun 28, 2022 |
| Grant date | Jun 28, 2022 |
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- Title
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- Abstract
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Abstract
Official abstract text for this publication.
This disclosure provides methods and compositions for treating lysosomal storage diseases in a subject. In one aspect of the invention, a transgene product is delivered to a subject by administering a recombinant neurotrophic viral vector containing the transgene to the brain. The viral vector delivers the transgene to a region of the brain which is susceptible to infection by the virus and which expresses the encoded recombinant viral gene product. Also provided are compositions for delivery of a transgene product to a subject by administering a recombinant neurotrophic viral vector containing the transgene to the subject's brain. The transgene product may be any that is deficient in a lysosomal storage disease.
First claim
Opening claim text (preview).
We claim: 1. A method to treat Niemann Pick A disease or Niemann Pick B disease in affected visceral organs in a subject, comprising directly injecting a therapeutically effective amount of a recombinant AAV4 or AAV5 viral vector comprising a therapeutic transgene encoding acid sphingomyelinase into at least one ventricle of the brain; wherein a therapeutic amount of the acid sphingomyelinase is expressed in the central nervous system and transported to affected visceral organs. 2. The method of claim 1 wherein the viral vector is administered by direct injection into a lateral ventricle of the brain. 3. The method of claim 1 wherein the viral vector is administered by direct injection into the fourth ventricle of the brain.
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Classifications
Viral vectors · CPC title
viral genome or elements thereof as genetic vector · CPC title
acting on glycosyl compounds (3.2), e.g. cellulases, lactases · CPC title
Glucosylceramidase (3.2.1.45), i.e. beta-glucocerebrosidase · CPC title
- A61K48/0075Primary
characterised by an aspect of the delivery route, e.g. oral, subcutaneous · CPC title
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Frequently asked questions
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- What does patent US11369693B2 cover?
- This disclosure provides methods and compositions for treating lysosomal storage diseases in a subject. In one aspect of the invention, a transgene product is delivered to a subject by administering a recombinant neurotrophic viral vector containing the transgene to the brain. The viral vector delivers the transgene to a region of the brain which is susceptible to infection by the virus and whi…
- Who is the assignee on this patent?
- Genzyme Corp
- What technology area does this patent fall under?
- Primary CPC classification A61K48/0075. Mapped technology areas include Human Necessities.
- When was this patent published?
- Publication date Tue Jun 28 2022 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 2 related publications on this page (citations in our corpus or others sharing the same primary CPC).