Expression vector for cholesterol 24-hydrolase in therapy of amyotrophic lateral sclerosis
US-2022054597-A1 · Feb 24, 2022 · US
Expression vector for cholesterol 24-hydrolase in therapy of polyglutamine repeat spinocerebellar ataxias
US12138298B2 · US · B2
| Field | Value |
|---|---|
| Publication number | US-12138298-B2 |
| Application number | US-201816480541-A |
| Country | US |
| Kind code | B2 |
| Filing date | Jan 30, 2018 |
| Priority date | Jan 30, 2017 |
| Publication date | Nov 12, 2024 |
| Grant date | Nov 12, 2024 |
How to read this patent
A practical reading order for non-experts. Skip the full description unless you need deep technical detail.
- Title
What the patent document calls the invention.
- Abstract
A short plain-language summary of the technical disclosure.
- Assignees and inventors
Who owns or filed the patent and who is credited as inventor.
- Key dates
Filing, priority, publication, and grant dates set the timeline.
- First independent claim
The legal scope of protection — read this for what is actually claimed.
- CPC / IPC classifications
Technology tags used to group this patent with similar filings.
- Citations and related patents
Prior art links and similar publications in this corpus.
Abstract
Official abstract text for this publication.
The present invention relates to a vector for use in the treatment of a polyglutamine repeat spinocerebellar ataxia, which vector comprises cholesterol 24-hydroxylase encoding nucleic acid.
First claim
Opening claim text (preview).
The invention claimed is: 1. A method of treatment of a Polyglutamine repeat spinocerebellar ataxia, comprising administering a vector which comprises cholesterol 24-hydroxylase encoding nucleic acid to a patient in need thereof. 2. The method of treatment of a Polyglutamine repeat spinocerebellar ataxia according to claim 1 , wherein the Polyglutamine repeat spinocerebellar ataxia is selected from the group of Spinocerebellar ataxia type 1 (SCA1), Spinocerebellar ataxia type 2 (SCA2), Spinocerebellar ataxia type 3 (SCA3), Spinocerebellar ataxia type 6 (SCA6), Spinocerebellar ataxia type 7 (SCA7) and Spinocerebellar ataxia type 17 (SCA17). 3. The method of treatment of a Polyglutamine repeat spinocerebellar ataxia according to claim 1 , wherein the Polyglutamine repeat spinocerebellar ataxia is Spinocerebellar ataxia type 3 (SCA3). 4. The method of treatment of a Polyglutamine repeat spinocerebellar ataxia according to claim 1 , wherein the vector comprises a nucleic acid sequence that encodes the amino acid sequence SEQ ID NO: 2. 5. The method of treatment of a Polyglutamine repeat spinocerebellar ataxia according to claim 1 , wherein the vector comprises the nucleic acid sequence SEQ ID NO: 1. 6. The method of treatment of a Polyglutamine repeat spinocerebellar ataxia according to claim 1 , wherein the vector is selected from the group of adenovirus, lentivirus, retrovirus, herpesvirus and Adeno-Associated Virus (AAV) vectors. 7. The method of treatment of a Polyglutamine repeat spinocerebellar ataxia according to claim 1 , wherein the vector is an AAV vector. 8. The method of treatment of a Polyglutamine repeat spinocerebellar ataxia according to claim 7 , wherein the vector is an AAV9 or AAV10 vector. 9. The method of treatment of a Polyglutamine repeat spinocerebellar ataxia according to claim 7 , wherein the vector is an AAVrh.10. 10. The method of treatment of a Polyglutamine repeat spinocerebellar ataxia according to claim 1 , wherein the vector is administered directly into the brain of the patient. 11. The method of treatment of a Polyglutamine repeat spinocerebellar ataxia according to claim 9 , wherein the vector is administered in at least a region of the brain selected from the group of cerebellum, brainstem, substantia nigra, striatum, frontotemporal lobes and visual cortex. 12. The method of treatment of a Polyglutamine repeat spinocerebellar ataxia according to claim 1 , wherein the vector is administered into the spinal cord of the patient. 13. The method of treatment of a Polyglutamine repeat spinocerebellar ataxia according to claim 1 , wherein the vector is administered by intravascular, intravenous, intranasal, intraventricular or intrathecal injection. 14. The method of treatment of a Polyglutamine repeat spinocerebellar ataxia according to claim 1 , comprising the administration of a pharmaceutical composition which comprises a therapeutically effective amount of the vector to the patient.
Assignees
Inventors
Classifications
Cholesterol 24-hydroxylase (1.14.13.98) · CPC title
Demonstrated in vivo effect · CPC title
viral genome or elements thereof as genetic vector · CPC title
Viral vectors · CPC title
Viruses; Bacteriophages; Compositions thereof; Preparation or purification thereof (preparing medicinal viral antigen or antibody compositions, e.g. virus vaccines, A61K39/00) · CPC title
Patent family
Related publications grouped by family.
External sources
Frequently asked questions
Answers are generated from the same data shown on this page.
- What does patent US12138298B2 cover?
- The present invention relates to a vector for use in the treatment of a polyglutamine repeat spinocerebellar ataxia, which vector comprises cholesterol 24-hydroxylase encoding nucleic acid.
- Who is the assignee on this patent?
- Brainvectis, Inst Nat Sante Rech Med, Univ De Coimbra, and 3 more
- What technology area does this patent fall under?
- Primary CPC classification A61K38/44. Mapped technology areas include Human Necessities.
- When was this patent published?
- Publication date Tue Nov 12 2024 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 3 related publications on this page (citations in our corpus or others sharing the same primary CPC).