Title:
IDENTIFICATION OF TISSUE-SPECIFIC EXTRAGENIC SAFE HARBORS FOR GENE THERAPY APPROACHES
Document Type and Number:
WIPO Patent Application WO/2023/212677
Kind Code:
A3
Abstract:
Compositions and methods for inserting a nucleic acid encoding a product of interest into a genomic safe harbor locus in a cell, a population of cells, or a subject or for expressing a nucleic acid encoding a product of interest from a genomic safe harbor locus in a cell, a population of cells, or a subject are provided. Also provided are cells or populations of cells comprising a nucleic acid construct comprising a coding sequence for a product of interest inserted into a genomic safe harbor locus. Also provided are methods of identifying genomic safe harbor loci for use in specific cell or tissue types.
Inventors:
BONETTI CIRO (US)
GONG GUOCHUN (US)
HE JING (US)
LIU JINRUI (US)
WARSHAW GREGG (US)
CHIAO ERIC (US)
ZAMBROWICZ BRIAN (US)
GONG GUOCHUN (US)
HE JING (US)
LIU JINRUI (US)
WARSHAW GREGG (US)
CHIAO ERIC (US)
ZAMBROWICZ BRIAN (US)
Application Number:
PCT/US2023/066343
Publication Date:
December 07, 2023
Filing Date:
April 28, 2023
Export Citation:
Assignee:
REGENERON PHARMA (US)
International Classes:
C12N15/85; A61K48/00; C12N9/22; C12N15/90
Domestic Patent References:
| WO2021055616A1 | 2021-03-25 | |||
| WO2021055592A1 | 2021-03-25 | |||
| WO2019169232A1 | 2019-09-06 | |||
| WO2016176191A1 | 2016-11-03 |
Other References:
WANG QINGNAN ET AL: "CRISPR-Cas9-Mediated In Vivo Gene Integration at the Albumin Locus Recovers Hemostasis in Neonatal and Adult Hemophilia B Mice", MOLECULAR THERAPY- METHODS & CLINICAL DEVELOPMENT, vol. 18, 1 September 2020 (2020-09-01), GB, pages 520 - 531, XP055942524, ISSN: 2329-0501, Retrieved from the Internet DOI: 10.1016/j.omtm.2020.06.025
MICHEL SADELAIN ET AL: "Safe harbours for the integration of new DNA in the human genome", NATURE REVIEWS CANCER, vol. 12, no. 1, 1 January 2011 (2011-01-01), pages 51 - 58, XP055018235, ISSN: 1474-175X, DOI: 10.1038/nrc3179
EIRINI P PAPAPETROU ET AL: "Gene Insertion Into Genomic Safe Harbors for Human Gene Therapy", MOLECULAR THERAPY, vol. 24, no. 4, 1 April 2016 (2016-04-01), US, pages 678 - 684, XP055547341, ISSN: 1525-0016, DOI: 10.1038/mt.2016.38
IRION S ET AL: "Identification and targeting of the ROSA26 locus in human embryonic stem cells", NATURE BIOTECHNOLOGY, NATURE PUBLISHING GROUP US, NEW YORK, vol. 25, no. 12, 1 December 2007 (2007-12-01), pages 1477 - 1482, XP008111005, ISSN: 1087-0156, [retrieved on 20071125], DOI: 10.1038/NBT1362
MICHEL SADELAIN ET AL: "Safe harbours for the integration of new DNA in the human genome", NATURE REVIEWS CANCER, vol. 12, no. 1, 1 January 2011 (2011-01-01), pages 51 - 58, XP055018235, ISSN: 1474-175X, DOI: 10.1038/nrc3179
EIRINI P PAPAPETROU ET AL: "Gene Insertion Into Genomic Safe Harbors for Human Gene Therapy", MOLECULAR THERAPY, vol. 24, no. 4, 1 April 2016 (2016-04-01), US, pages 678 - 684, XP055547341, ISSN: 1525-0016, DOI: 10.1038/mt.2016.38
IRION S ET AL: "Identification and targeting of the ROSA26 locus in human embryonic stem cells", NATURE BIOTECHNOLOGY, NATURE PUBLISHING GROUP US, NEW YORK, vol. 25, no. 12, 1 December 2007 (2007-12-01), pages 1477 - 1482, XP008111005, ISSN: 1087-0156, [retrieved on 20071125], DOI: 10.1038/NBT1362
Attorney, Agent or Firm:
SCHILLING, Stephen H. et al. (US)
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