Invention Grant
- Patent Title: Compositions and methods for transient delivery of nucleases
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Application No.: US15550452Application Date: 2016-02-12
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Publication No.: US10584321B2Publication Date: 2020-03-10
- Inventor: Guangping Gao , Phillip D. Zamore , Dan Wang
- Applicant: University of Massachusetts
- Applicant Address: US MA Boston
- Assignee: University of Massachusetts
- Current Assignee: University of Massachusetts
- Current Assignee Address: US MA Boston
- Agency: Wolf, Greenfield & Sacks, P.C.
- International Application: PCT/US2016/017886 WO 20160212
- International Announcement: WO2016/131009 WO 20160818
- Main IPC: C12N9/22
- IPC: C12N9/22 ; A61K48/00 ; C12N15/90 ; C07K14/005 ; C12N7/00 ; C12N15/11 ; A61K38/00

Abstract:
The disclosure in some aspects relates to recombinant adeno-associated viruses having nuclease grafted to one or more capsid proteins. In some aspects, the disclosure relates to isolated AAV capsid proteins having terminally grafted nucleases and isolated nucleic acids encoding the same. Recent approaches to delivering nucleases to cells for gene editing have focused on delivering of expression vectors engineered to express the nucleases in target cells. However, these approaches have proved to be problematic in many instances due to genotoxicity resulting from to prolonged expression of gene editing system in vivo. To prevent such off-target genotoxicity due to prolonged presence of a gene editing system, several studies explored delivery of mRNA or protein instead of delivering the gene coding for the nucleases in cell culture.
Public/Granted literature
- US20180179501A9 COMPOSITIONS AND METHODS FOR TRANSIENT DELIVERY OF NUCLEASES Public/Granted day:2018-06-28
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