Login| Sign Up| Help| Contact|

Patent Searching and Data


Title:
GENE EDITING FOR AUTOSOMAL DOMINANT DISEASES
Document Type and Number:
WIPO Patent Application WO/2019/183630
Kind Code:
A3
Abstract:
The present disclosure provides methods for treating autosomal dominant diseases in a subject. In some aspects, the methods involve the use of a gene editing enzyme with a pair of unique guide RNA sequences that targets both mutant and wildtype forms of autosomal dominant disease-related gene for destruction in cells, and then supplying the cells with wildtype autosomal dominant disease-related gene cDNA which is codon modified to evade recognition by the guide RNAs. These methods are broadly applicable to any autosomal dominant disease.

More Like This:
JP2006516288Aptamer remedies useful in eye medications
JP2017536366LNA chiral phosphorothioate
WO/2005/055945MUCOADHESIVE DRUG DELIVERY DEVICES AND METHODS OF MAKING AND USING THEREOF
Inventors:
WU WEN-HSUAN (US)
TSAI YI-TING (US)
TSANG STEPHEN (US)
Application Number:
PCT/US2019/023881
Publication Date:
November 21, 2019
Filing Date:
March 25, 2019
Export Citation:
Click for automatic bibliography generation   Help
Assignee:
UNIV COLUMBIA (US)
International Classes:
A61K48/00; C12N15/09; C12N15/63; C12N15/86; C12N15/861
Domestic Patent References:
WO2016176690A22016-11-03
WO2016186772A22016-11-24
WO2016148994A12016-09-22
Foreign References:
US20160097061A12016-04-07
US20060105364A12006-05-18
US20170173183A12017-06-22
US20110052602A12011-03-03
US20140023702A12014-01-23
US20120289582A12012-11-15
Attorney, Agent or Firm:
DAVITZ, Michael A. et al. (US)
Download PDF:
View/Download PDF      PDF Help



 
Previous Patent: CRYPTOGRAPHICALLY ENFORCED DATA EXCHANGE

Next Patent: PACKING MATERIAL CARTRIDGE