Title:
DIFFERENTIAL KNOCKOUT OF A HETEROZYGOUS ALLELE OF SAMD9
Document Type and Number:
WIPO Patent Application WO/2021/243218
Kind Code:
A3
Abstract:
RNA molecules comprising a guide sequence portion having 17-50 contiguous nucleotides containing nucleotides in the sequence set forth in any one of SEQ ID NOs: 1-20640 and compositions, methods, and uses thereof.
Inventors:
EMMANUEL RAFI (IL)
DICKEN JOSEPH (IL)
GOLAN MASHIACH MICHAL (IL)
DICKEN JOSEPH (IL)
GOLAN MASHIACH MICHAL (IL)
Application Number:
PCT/US2021/034873
Publication Date:
February 17, 2022
Filing Date:
May 28, 2021
Export Citation:
Assignee:
EMENDOBIO INC (US)
EMMANUEL RAFI (IL)
DICKEN JOSEPH (IL)
GOLAN MASHIACH MICHAL (IL)
EMMANUEL RAFI (IL)
DICKEN JOSEPH (IL)
GOLAN MASHIACH MICHAL (IL)
International Classes:
A61K48/00; A61K38/46; C12N15/63; C12N15/90
Domestic Patent References:
| WO2015048577A2 | 2015-04-02 |
Foreign References:
| US20180291383A1 | 2018-10-11 | |||
| US20100209914A1 | 2010-08-19 |
Other References:
ONUMA SHINSUKE, WADA TAMAKI, ARAKI RYOSUKE, WADA KAZUKO, TANASE-NAKAO KANAKO, NARUMI SATOSHI, FUKUI MIHO, SHOJI YASUKO, ETANI YURI: "MIRAGE syndrome caused by a novel missense variant (p.Ala1479Ser) in the SAMD9 gene", HUMAN GENOME VARIATION, vol. 7, no. 1, 1 December 2020 (2020-12-01), XP055905633, DOI: 10.1038/s41439-020-0091-5
GIL-VAREA ELIA, URCELAY ELENA, VILARIÑO-GÜELL CARLES, COSTA CARME, MIDAGLIA LUCIANA, MATESANZ FUENCISLA, RODRÍGUEZ-ANTIGÜEDAD ALFR: "Exome sequencing study in patients with multiple sclerosis reveals variants associated with disease course", JOURNAL OF NEUROINFLAMMATION, vol. 15, no. 1, 1 December 2018 (2018-12-01), pages 265, XP055905637, DOI: 10.1186/s12974-018-1307-1
NOUNAMO, B ET AL.: "An interaction domain in human SAMD9 is essential for myxoma virus host-range determinant M062 antagonism of host anti-viral function", VIROLOGY, vol. 503, 31 January 2017 (2017-01-31), pages 94 - 102, XP029929104, DOI: 10.1016/j.virol. 2017.01.00 4
VIAENE ANGELA N, HARDING BRIAN N: "The Neuropathology of MIRAGE Syndrome", JOURNAL OF NEUROPATHOLOGY AND EXPERIMENTAL NEUROLOGY, LIPPINCOTT WILLIAMS AND WILKINS., NEW YORK, NY., vol. 79, no. 4, 1 April 2020 (2020-04-01), NEW YORK, NY. , pages 458 - 462, XP055905639, ISSN: 0022-3069, DOI: 10.1093/jnen/nlaa009
GIL-VAREA ELIA, URCELAY ELENA, VILARIÑO-GÜELL CARLES, COSTA CARME, MIDAGLIA LUCIANA, MATESANZ FUENCISLA, RODRÍGUEZ-ANTIGÜEDAD ALFR: "Exome sequencing study in patients with multiple sclerosis reveals variants associated with disease course", JOURNAL OF NEUROINFLAMMATION, vol. 15, no. 1, 1 December 2018 (2018-12-01), pages 265, XP055905637, DOI: 10.1186/s12974-018-1307-1
NOUNAMO, B ET AL.: "An interaction domain in human SAMD9 is essential for myxoma virus host-range determinant M062 antagonism of host anti-viral function", VIROLOGY, vol. 503, 31 January 2017 (2017-01-31), pages 94 - 102, XP029929104, DOI: 10.1016/j.virol. 2017.01.00 4
VIAENE ANGELA N, HARDING BRIAN N: "The Neuropathology of MIRAGE Syndrome", JOURNAL OF NEUROPATHOLOGY AND EXPERIMENTAL NEUROLOGY, LIPPINCOTT WILLIAMS AND WILKINS., NEW YORK, NY., vol. 79, no. 4, 1 April 2020 (2020-04-01), NEW YORK, NY. , pages 458 - 462, XP055905639, ISSN: 0022-3069, DOI: 10.1093/jnen/nlaa009
Attorney, Agent or Firm:
GERSHIK, Gary, J. (US)
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