Methods and compositions for treating hemophilia
US-9771403-B2 · Sep 26, 2017 · US
Methods and compositions for genome engineering
US10968261B2 · US · B2
| Field | Value |
|---|---|
| Publication number | US-10968261-B2 |
| Application number | US-201816107891-A |
| Country | US |
| Kind code | B2 |
| Filing date | Aug 21, 2018 |
| Priority date | Dec 9, 2013 |
| Publication date | Apr 6, 2021 |
| Grant date | Apr 6, 2021 |
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- Title
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Abstract
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Disclosed herein are methods and compositions for insertion of transgene sequences encoding proteins that is aberrantly expressed in disease or disorder such as a lysosomal storage disease.
First claim
Opening claim text (preview).
What is claimed is: 1. A cell comprising a pair of zinc finger nucleases (ZFNs) that cleave an endogenous albumin gene, each zinc finger nuclease comprising a cleavage domain and a zinc finger protein (ZFP) that binds to a target site in the endogenous albumin gene, wherein the zinc finger proteins of the pair are selected from the group consisting of a left ZFP designated SBS# 47171, 47192, 47863, or 47864, the left ZFP comprising five recognition helix regions ordered F1 to F5 as shown in a single row of the following Table: SBS# F1 F2 F3 F4 F5 47171 QSGNLSR LKQNLCM WADNLQN TSGNLTR RQSHLCL (SEQ ID (SEQ ID (SEQ ID (SEQ ID (SEQ ID NO: 23) NO: 18) NO: 24) NO: 20) NO: 21) 47863 QSGNLAR LIQYLQS WADNLQN TSGNLTR RQSHLSL (SEQ ID (SEQ ID (SEQ ID (SEQ ID (SEQ ID NO: 2) NO: 26) NO: 24) NO: 20) NO: 27) 47192 QSGNLAR LIQYLQS WADNLQN TSGNLTR RQSHLCL (SEQ ID (SEQ ID (SEQ ID (SEQ ID (SEQ ID NO: 2) NO: 26) NO: 24) NO: 20) NO: 21) 47864 QSGNLAR LIQYLQS WQSNLQN TSGNLTR RQSHLCL (SEQ ID (SEQ ID (SEQ ID (SEQ ID (SEQ ID NO: 2) NO: 26) NO: 19) NO: 20) NO: 21) and a right ZFP designated SBS# 40477, 47079, 47169, 47898 or 47931, the right ZFP comprising six recognition helix regions ordered F1 to F6 as shown in a single row of the following Table: SBS# F1 F2 F3 47931 TPQLLDR LKWNLRT DQSNLNA (SEQ ID (SEQ ID (SEQ ID NO: 14) NO: 9) NO: 25) 47079 TPQLLDR LKWNLRT DQSNLRA (SEQ ID (SEQ ID (SEQ ID NO: 14) NO: 9) NO: 10) 47898 TPQLLDR LKHNLLT DQSNLNA (SEQ ID (SEQ ID (SEQ ID NO: 14) NO: 28 NO: 25) 47169 TPQLLDR LKWNLRT DQSNLRA (SEQ ID (SEQ ID (SEQ ID NO: 14) NO: 9) NO: 10) 40477 QSSDLSR LKHNLLT LKHNLLT (SEQ ID (SEQ ID (SEQ ID NO: 8) NO: 28) NO: 28) SBS# F4 F5 F6 47931 RNFSLTM LRHDLDR HRSNLNK (SEQ ID (SEQ ID (SEQ ID NO: 15) NO: 16) NO: 12) 47079 RNFSLTM LRHDLDR HRSNLNK (SEQ ID (SEQ ID (SEQ ID NO: 15) NO: 16) NO: 12)
Assignees
Inventors
Classifications
viral genome or elements thereof as genetic vector · CPC title
Antihaemorrhagics; Procoagulants; Haemostatic agents; Antifibrinolytic agents · CPC title
Ribonucleases {[RNase]; Deoxyribonucleases [DNase]} · CPC title
Viral vectors · CPC title
Coagulation factor IXa (3.4.21.22) · CPC title
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- What does patent US10968261B2 cover?
- Disclosed herein are methods and compositions for insertion of transgene sequences encoding proteins that is aberrantly expressed in disease or disorder such as a lysosomal storage disease.
- Who is the assignee on this patent?
- Sangamo Therapeutics Inc
- What technology area does this patent fall under?
- Primary CPC classification C07K14/47. Mapped technology areas include Chemistry & Metallurgy.
- When was this patent published?
- Publication date Tue Apr 06 2021 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).