THERAPEUTIC USES OF GENOME EDITING WITH CRISPR/Cas SYSTEMS
US-2015071889-A1 · Mar 12, 2015 · US
Methods and compositions for treatment of a genetic condition
US9902974B2 · US · B2
| Field | Value |
|---|---|
| Publication number | US-9902974-B2 |
| Application number | US-201514965629-A |
| Country | US |
| Kind code | B2 |
| Filing date | Dec 10, 2015 |
| Priority date | May 15, 2013 |
| Publication date | Feb 27, 2018 |
| Grant date | Feb 27, 2018 |
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- Title
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Abstract
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Methods and compositions comprising a fusion protein comprising a Cas protein and a functional domain in association with a single guide RNA targeted to an endogenous gene for genetic alteration of cells are provided.
First claim
Opening claim text (preview).
What is claimed is: 1. A fusion protein comprising a Cas protein and a functional domain in association with a single-guide RNA comprising a sequence selected from the group consisting of SEQ ID NOs:149-158 and 167-215. 2. The fusion protein of claim 1 , wherein the functional domain is a transcriptional activation domain, a transcriptional repression domain or a nuclease domain. 3. The fusion protein of claim 2 , wherein the nuclease domain is a FokI endonuclease domain. 4. The fusion protein of claim 3 , wherein the FokI endonuclease domain comprises one or more mutations in the dimerization domain. 5. A dimer comprising first and second fusion proteins according to claim 3 . 6. The dimer of claim 5 , wherein the first fusion protein comprises a FokI endonuclease domain comprising one or more mutations in the catalytic domain. 7. A pharmaceutical composition comprising a fusion protein according to claim 1 .
Assignees
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Classifications
containing a Zn-finger domain for DNA binding · CPC title
Ribonucleases {[RNase]; Deoxyribonucleases [DNase]} · CPC title
- C12N15/907Primary
in mammalian cells · CPC title
Type II site-specific deoxyribonuclease (3.1.21.4) · CPC title
Special therapeutic applications · CPC title
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Frequently asked questions
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- What does patent US9902974B2 cover?
- Methods and compositions comprising a fusion protein comprising a Cas protein and a functional domain in association with a single guide RNA targeted to an endogenous gene for genetic alteration of cells are provided.
- Who is the assignee on this patent?
- Sangamo Biosciences Inc, Sangamo Therapeutics Inc
- What technology area does this patent fall under?
- Primary CPC classification C12N15/907. Mapped technology areas include Chemistry & Metallurgy.
- When was this patent published?
- Publication date Tue Feb 27 2018 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 1 related publication on this page (citations in our corpus or others sharing the same primary CPC).