Title:
METHODS AND SYSTEMS FOR DETERMINING EDITING OUTCOMES FROM REPAIR OF TARGETED ENDONUCLEASE MEDIATED CUTS
Document Type and Number:
WIPO Patent Application WO/2019/232494
Kind Code:
A3
Abstract:
The present disclosure provides a method for determining the probability distribution of editing outcomes, such as indel length or genotype, resulting from a targeted endonuclease mediated edit, as well as a method for determining a guide RNAs or donor polynucleotides able to produce a desired editing outcome. The editing outcomes can be cell-type specific. The methods can comprise the use of machine learning models to produce the probability distributions.
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Inventors:
CONANT DAVID (US)
HSIAU TIMOTHY (US)
STONER RICHARD (US)
HSIAU TIMOTHY (US)
STONER RICHARD (US)
Application Number:
PCT/US2019/035079
Publication Date:
January 09, 2020
Filing Date:
May 31, 2019
Export Citation:
Assignee:
SYNTHEGO CORP (US)
International Classes:
C12Q1/68
Domestic Patent References:
| WO2016057961A1 | 2016-04-14 | |||
| WO2017053879A1 | 2017-03-30 | |||
| WO2016183402A2 | 2016-11-17 | |||
| WO2017081097A1 | 2017-05-18 |
Foreign References:
| US20140186843A1 | 2014-07-03 | |||
| US20060074824A1 | 2006-04-06 | |||
| US20180020646A1 | 2018-01-25 |
Other References:
WANG ET AL.: "CRISPR-DAV: CRISPR NGS Data Analysis and Visualization Pipeline", BIOINFORMATICS, vol. 33, no. 23, 14 August 2017 (2017-08-14), pages 3811 - 3812, XP055672196
LIANG ET AL.: "Enhanced CRISPR/Cas9-Mediated Precise Genome Editing by Improved Design and Delivery of gRNA, Cas9 Nuclease, and Donor DNA", JOURNAL OF BIOTECHNOLOGY, vol. 241, 11 November 2016 (2016-11-11), pages 136 - 146, XP029860705, DOI: 10.1016/j.jbiotec.2016.11.011
SURUN ET AL.: "High Efficiency Gene Correction in Hematopoietic Cells by Donor-Template- Free CRISPR/Cas9 Genome Editing", MOLECULAR THERAPY: NUCLEIC ACIDS, vol. 10, 10 November 2017 (2017-11-10), pages 1 - 8, XP055606586, DOI: 10.1016/j.omtn.2017.11.001
LIANG ET AL.: "Enhanced CRISPR/Cas9-Mediated Precise Genome Editing by Improved Design and Delivery of gRNA, Cas9 Nuclease, and Donor DNA", JOURNAL OF BIOTECHNOLOGY, vol. 241, 11 November 2016 (2016-11-11), pages 136 - 146, XP029860705, DOI: 10.1016/j.jbiotec.2016.11.011
SURUN ET AL.: "High Efficiency Gene Correction in Hematopoietic Cells by Donor-Template- Free CRISPR/Cas9 Genome Editing", MOLECULAR THERAPY: NUCLEIC ACIDS, vol. 10, 10 November 2017 (2017-11-10), pages 1 - 8, XP055606586, DOI: 10.1016/j.omtn.2017.11.001
Attorney, Agent or Firm:
ROHATGI, Kyanne R. (US)
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