Title:
MUSCLE-SPECIFIC REGULATORY CASSETTES
Document Type and Number:
WIPO Patent Application WO/2024/020444
Kind Code:
A3
Abstract:
The present disclosure relates to nucleic acid regulatory cassettes useful for the expression of transgene payloads in specific muscle tissues. Also included in the present disclosure are vectors, including adeno-associated virus (AAV) vectors, comprising the regulatory cassettes of the invention, as well as compositions and methods of treating genetic diseases or disorders also comprising said regulatory cassettes.
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Inventors:
JONES PETER L (US)
HIMEDA CHARIS L (US)
HIMEDA CHARIS L (US)
Application Number:
PCT/US2023/070498
Publication Date:
March 28, 2024
Filing Date:
July 19, 2023
Export Citation:
Assignee:
NEVADA RES & INNOVATION CORPORATION (US)
UNIV NEVADA RENO (US)
UNIV NEVADA RENO (US)
International Classes:
A61K48/00; A61P21/00; C12N15/113; C12N15/864; C07H21/04
Domestic Patent References:
| WO2022008711A1 | 2022-01-13 | |||
| WO2021211325A1 | 2021-10-21 |
Other References:
HIMEDA CHARIS L., JONES TAKAKO I., JONES PETER L.: "Targeted epigenetic repression by CRISPR/dSaCas9 suppresses pathogenic DUX4-fl expression in FSHD", MOLECULAR THERAPY- METHODS & CLINICAL DEVELOPMENT, vol. 20, 1 March 2021 (2021-03-01), GB , pages 298 - 311, XP055865179, ISSN: 2329-0501, DOI: 10.1016/j.omtm.2020.12.001
QUYNH-GIAO V. NGUYEN, JEAN N. BUSKIN, CHARIS L. HIMEDA, MARGARET A. SHIELD, STEPHEN D. HAUSCHKA: "Differences in the Function of Three Conserved E-boxes of the Muscle Creatine Kinase Gene in Cultured Myocytes and in Transgenic Mouse Skeletal and Cardiac Muscle", JOURNAL OF BIOLOGICAL CHEMISTRY, vol. 278, no. 47, 1 November 2003 (2003-11-01), US , pages 46494 - 46505, XP093154765, ISSN: 0021-9258, DOI: 10.1074/jbc.M308194200
MAJA Z SALVA, CHARIS L HIMEDA, PHILLIP WL TAI, EIKO NISHIUCHI, PAUL GREGOREVIC, JAMES M ALLEN, ERIC E FINN, QUYNH G NGUYEN, MICHAE: "Design of Tissue-specific Regulatory Cassettes for High-level rAAV-mediated Expression in Skeletal and Cardiac Muscle", MOLECULAR THERAPY, vol. 15, no. 2, 1 February 2007 (2007-02-01), pages 320 - 329, XP055078517, ISSN: 15250016, DOI: 10.1038/sj.mt.6300027
QUYNH-GIAO V. NGUYEN, JEAN N. BUSKIN, CHARIS L. HIMEDA, MARGARET A. SHIELD, STEPHEN D. HAUSCHKA: "Differences in the Function of Three Conserved E-boxes of the Muscle Creatine Kinase Gene in Cultured Myocytes and in Transgenic Mouse Skeletal and Cardiac Muscle", JOURNAL OF BIOLOGICAL CHEMISTRY, vol. 278, no. 47, 1 November 2003 (2003-11-01), US , pages 46494 - 46505, XP093154765, ISSN: 0021-9258, DOI: 10.1074/jbc.M308194200
MAJA Z SALVA, CHARIS L HIMEDA, PHILLIP WL TAI, EIKO NISHIUCHI, PAUL GREGOREVIC, JAMES M ALLEN, ERIC E FINN, QUYNH G NGUYEN, MICHAE: "Design of Tissue-specific Regulatory Cassettes for High-level rAAV-mediated Expression in Skeletal and Cardiac Muscle", MOLECULAR THERAPY, vol. 15, no. 2, 1 February 2007 (2007-02-01), pages 320 - 329, XP055078517, ISSN: 15250016, DOI: 10.1038/sj.mt.6300027
Attorney, Agent or Firm:
CORLESS, Peter, F. et al. (US)
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