Title:
METHODS AND COMPOSITIONS UTILIZING CPF1 FOR RNA-GUIDED GENE EDITING
Document Type and Number:
WIPO Patent Application WO/2017/066588
Kind Code:
A3
Abstract:
Compositions include endonucleases of the family Cpf1 (CRISPR from Prevotella and Francisella 1); and at least one guide RNA (gRNA) complementary to a target sequence in a gene to specifically guide the Cpf1 endonuclease to the target site in a host cell in vitro or in vivo. Methods of treating a subject include the use of one or more of these compositions.
Inventors:
KHALILI KAMEL (US)
MALCOLM THOMAS (US)
MALCOLM THOMAS (US)
Application Number:
PCT/US2016/057069
Publication Date:
June 08, 2017
Filing Date:
October 14, 2016
Export Citation:
Assignee:
TEMPLE UNIV - OF THE COMMONWEALTH SYSTEM OF HIGHER EDUCATION (US)
International Classes:
A61K48/00; C12N15/09; C12N15/113; C12N15/63
Domestic Patent References:
| WO2015031775A1 | 2015-03-05 | |||
| WO2014165349A1 | 2014-10-09 | |||
| WO2014191128A1 | 2014-12-04 |
Other References:
ZETSCHE, B ET AL.: "Cpf1 Is a Single RNA-Guided Endonuclease of a Class 2 CRISPR-Cas System.", CELL., vol. 22, no. 3, 25 September 2015 (2015-09-25), pages 759 - 771, XP055267511
Attorney, Agent or Firm:
ZACHARIADES, Nicholas, A. et al. (US)
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