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CNS targeting AAV vectors and methods of use thereof
US9701984B2 · US · B2
| Field | Value |
|---|---|
| Publication number | US-9701984-B2 |
| Application number | US-201414445670-A |
| Country | US |
| Kind code | B2 |
| Filing date | Jul 29, 2014 |
| Priority date | Apr 23, 2010 |
| Publication date | Jul 11, 2017 |
| Grant date | Jul 11, 2017 |
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- Title
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- Abstract
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Abstract
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The invention in some aspects relates to recombinant adeno-associated viruses useful for targeting transgenes to CNS tissue, and compositions comprising the same, and methods of use thereof. In some aspects, the invention provides methods and compositions for treating CNS-related disorders.
First claim
Opening claim text (preview).
What is claimed is: 1. A method for delivering a transgene to CNS tissue in a subject, the method comprising: administering an effective amount of a rAAV comprising a promoter operably linked with a transgene to central nervous system (CNS) tissue by intrathecal administration, wherein the rAAV infects cells of the CNS of the subject, wherein the transgene encodes an inhibitory RNA that has the sequence of SEQ ID NO: 26, wherein the inhibitory RNA is complementary with and specifically binds to a target site sequence of the SOD1 mRNA and inhibits expression of SOD1 protein in the subject, wherein the target site sequence of the SOD1 mRNA comprises at least 5 nucleotides that are complementary with a sequence as set forth in SEQ ID NO: 26. 2. The method of claim 1 , wherein the intrathecal administration is in the lumbar region of the subject. 3. The method of claim 1 , wherein the dose of the rAAV for intrathecal administration is in a range of 10 10 genome copies to 10 14 genome copies. 4. The method of claim 1 , wherein the intrathecal administration is in the cervical region of the subject. 5. The method of claim 1 , wherein the intrathecal administration is in the thoracic region of the subject. 6. The method of claim 1 , wherein the cells of CNS tissue are oligodendrocytes, astrocytes, neurons, brain parenchyma cells, and/or Purkinje cells. 7. The method of claim 1 , wherein the inhibitory RNA is an antisense RNA, a shRNA or a miRNA.
Assignees
Inventors
Classifications
characterised by an aspect of the delivery route, e.g. oral, subcutaneous · CPC title
Special targeting system for viral vectors · CPC title
- C12N15/86Primary
Viral vectors · CPC title
by genetic engineering · CPC title
acting on carbon-nitrogen bonds, other than peptide bonds (3.5), e.g. asparaginase · CPC title
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Frequently asked questions
Answers are generated from the same data shown on this page.
- What does patent US9701984B2 cover?
- The invention in some aspects relates to recombinant adeno-associated viruses useful for targeting transgenes to CNS tissue, and compositions comprising the same, and methods of use thereof. In some aspects, the invention provides methods and compositions for treating CNS-related disorders.
- Who is the assignee on this patent?
- Univ Massachusetts
- What technology area does this patent fall under?
- Primary CPC classification C12N15/86. Mapped technology areas include Chemistry & Metallurgy.
- When was this patent published?
- Publication date Tue Jul 11 2017 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 4 related publications on this page (citations in our corpus or others sharing the same primary CPC).