Title:
PDX DMD MOUSE MODEL, METHOD FOR CONSTRUCTING THE SAME AND USE THEREOF
Document Type and Number:
WIPO Patent Application WO/2022/109890
Kind Code:
A1
Abstract:
Provided are a method for constructing a PDX DMD mouse model, a PDX DMD mouse model, the use of the PDX DMD mouse model, and a genome editing method for restoring dystrophin expression in DMD patient muscle fibers.
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Inventors:
LI XIAOPING (CN)
XIANG PENG (CN)
LI WEIQIANG (CN)
KE QIONG (CN)
XIANG PENG (CN)
LI WEIQIANG (CN)
KE QIONG (CN)
Application Number:
PCT/CN2020/131620
Publication Date:
June 02, 2022
Filing Date:
November 26, 2020
Export Citation:
Assignee:
UNIV SUN YAT SEN (CN)
International Classes:
A01K67/027; A01K67/02; A61K48/00; C12N5/10; C12N15/90
Domestic Patent References:
| WO2017072590A1 | 2017-05-04 |
Foreign References:
| CN109863241A | 2019-06-07 | |||
| CN106714845A | 2017-05-24 |
Other References:
MENG JINHONG, MUNTONI FRANCESCO, MORGAN JENNIFER: "CD133+ cells derived from skeletal muscles of Duchenne muscular dystrophy patients have a compromised myogenic and muscle regenerative capability", STEM CELL RESEARCH, vol. 30, 1 July 2018 (2018-07-01), NL , pages 43 - 52, XP055932353, ISSN: 1873-5061, DOI: 10.1016/j.scr.2018.05.004
MOTOHASHI，N. ET AL.: "Isolation， culture， and transplantation of muscle satellite cells.", J VIS EXP., vol. 86, 8 April 2014 (2014-04-08)
MENG JINHONG, CHUN SOYON, ASFAHANI ROWAN, LOCHMÜLLER HANNS, MUNTONI FRANCESCO, MORGAN JENNIFER: "Human Skeletal Muscle–derived CD133+ Cells Form Functional Satellite Cells After Intramuscular Transplantation in Immunodeficient Host Mice", MOLECULAR THERAPY, vol. 22, no. 5, 1 May 2014 (2014-05-01), US , pages 1008 - 1017, XP055932355, ISSN: 1525-0016, DOI: 10.1038/mt.2014.26
TORRENTE Y; BELICCHI M; MARCHESI C; D'ANTONA G; COGIAMANIAN F; PISATI F; GAVINA M; GIORDANO R; TONLORENZI R; FAGIOLARI G; LAMPERTI: "Autologous transplantation of muscle-derived CD133+ stem cells in Duchenne muscle patients.", CELL TRANSPLANTATION, vol. 16, no. 6, 1 January 2007 (2007-01-01), US , pages 563 - 577, XP009193750, ISSN: 0963-6897, DOI: 10.3727/000000007783465064
BENCHAOUIR R; MEREGALLI M; FARINI A; D'ANTONA G; BELICCHI M; GOYENVALLE A; BATTISTELLI M; BRESOLIN N; BOTTINELLI R; GARCIA L; TORR: "Restoration of Human Dystrophin Following Transplantation of Exon-Skipping-Engineered DMD Patient Stem Cells into Dystrophic Mice", CELL STEM CELL, ELSEVIER, vol. 1, no. 6, 13 December 2007 (2007-12-13), AMSTERDAM, NL , pages 646 - 657, XP009107565, ISSN: 1934-5909, DOI: 10.1016/j.ste.2007.09.016
ZHANG SHU;WU SHI-WEN: "Therapeutic progress of Duchenne muscular dystrophy", PRACTICAL PHARMACY AND CLINICAL REMEDIES, vol. 22, no. 9, 26 September 2019 (2019-09-26), pages 897 - 903, XP055932361, DOI: 10.14053/j.cnki.ppcr.201909001
MOTOHASHI，N. ET AL.: "Isolation， culture， and transplantation of muscle satellite cells.", J VIS EXP., vol. 86, 8 April 2014 (2014-04-08)
MENG JINHONG, CHUN SOYON, ASFAHANI ROWAN, LOCHMÜLLER HANNS, MUNTONI FRANCESCO, MORGAN JENNIFER: "Human Skeletal Muscle–derived CD133+ Cells Form Functional Satellite Cells After Intramuscular Transplantation in Immunodeficient Host Mice", MOLECULAR THERAPY, vol. 22, no. 5, 1 May 2014 (2014-05-01), US , pages 1008 - 1017, XP055932355, ISSN: 1525-0016, DOI: 10.1038/mt.2014.26
TORRENTE Y; BELICCHI M; MARCHESI C; D'ANTONA G; COGIAMANIAN F; PISATI F; GAVINA M; GIORDANO R; TONLORENZI R; FAGIOLARI G; LAMPERTI: "Autologous transplantation of muscle-derived CD133+ stem cells in Duchenne muscle patients.", CELL TRANSPLANTATION, vol. 16, no. 6, 1 January 2007 (2007-01-01), US , pages 563 - 577, XP009193750, ISSN: 0963-6897, DOI: 10.3727/000000007783465064
BENCHAOUIR R; MEREGALLI M; FARINI A; D'ANTONA G; BELICCHI M; GOYENVALLE A; BATTISTELLI M; BRESOLIN N; BOTTINELLI R; GARCIA L; TORR: "Restoration of Human Dystrophin Following Transplantation of Exon-Skipping-Engineered DMD Patient Stem Cells into Dystrophic Mice", CELL STEM CELL, ELSEVIER, vol. 1, no. 6, 13 December 2007 (2007-12-13), AMSTERDAM, NL , pages 646 - 657, XP009107565, ISSN: 1934-5909, DOI: 10.1016/j.ste.2007.09.016
ZHANG SHU;WU SHI-WEN: "Therapeutic progress of Duchenne muscular dystrophy", PRACTICAL PHARMACY AND CLINICAL REMEDIES, vol. 22, no. 9, 26 September 2019 (2019-09-26), pages 897 - 903, XP055932361, DOI: 10.14053/j.cnki.ppcr.201909001
Attorney, Agent or Firm:
GUANGZHOU BANGZHUAN GAOZHI INTELLECTUAL PROPERTY AGENCY (SPECIAL GENERAL PARTNERSHIP) (CN)
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