Delivery methods and compositions for nuclease-mediated genome engineering in hematopoietic stem cells
US-2015174169-A1 · Jun 25, 2015 · US
Method of treating factor IX deficiency using nuclease-mediated targeted integration
US10370680B2 · US · B2
| Field | Value |
|---|---|
| Publication number | US-10370680-B2 |
| Application number | US-201514630128-A |
| Country | US |
| Kind code | B2 |
| Filing date | Feb 24, 2015 |
| Priority date | Feb 24, 2014 |
| Publication date | Aug 6, 2019 |
| Grant date | Aug 6, 2019 |
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Abstract
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Disclosed herein are methods and compositions for targeted, nuclease-mediated insertion of transgene sequences into the genome of a cell.
First claim
Opening claim text (preview).
What is claimed: 1. A method for treating a Factor IX (F.IX) deficiency in a mammal, the method comprising: (i) intravenously administering one or more adeno-associated viral (AAV) vectors encoding a pair of zinc finger nucleases (ZFNs) or a pair of TAL-effector protein nucleases (TALENs) into a mammal with a F.IX deficiency such that a double-stranded break in an endogenous albumin gene occurs in liver cells of the mammal, and (ii) intravenously administering one or more AAV vectors comprising a donor sequence comprising a transgene encoding a functional F.IX protein into the mammal 1 to 3 days after administering the one or more AAV vectors encoding the pair of ZFNs or TALENs, wherein the transgene is flanked by nucleic acid sequences homologous to the albumin gene, such that the transgene is integrated into the endogenous albumin gene, the functional F.IX protein is expressed in liver cells of the mammal at increased levels as compared to mammals in which the AAV vectors of (i) and (ii) are administered on the same day. 2. The method of claim 1 , wherein expression of the transgene is driven by an endogenous albumin promoter. 3. The method of claim 1 , wherein the F.IX protein is a is human F.IX (hF .IX) protein.
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Classifications
Drugs for specific purposes, not provided for in groups A61P1/00-A61P41/00 · CPC title
Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents · CPC title
- C12N15/907Primary
in mammalian cells · CPC title
Use of virus, viral particle or viral elements as a vector · CPC title
for animal cells · CPC title
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- What does patent US10370680B2 cover?
- Disclosed herein are methods and compositions for targeted, nuclease-mediated insertion of transgene sequences into the genome of a cell.
- 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 Aug 06 2019 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).