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Title:
希少疾患の処置のための方法および組成物
Document Type and Number:
Japanese Patent JP7381476
Kind Code:
B2
Abstract:
The present disclosure is in the field of modulation of genes involved in rare diseases including for diagnostics and therapeutics for rare diseases such as Angelman's Syndrome, Facioscapulohumeral Muscular Dystrophy (FHMD), Amyotrophic Lateral Sclerosis (ALS), Frontotemporal dementia (FTD) and Spinal Muscular Atrophy (SMA).

Inventors:
Michael Sea Holmes
Bridget e-Riley
Thomas Wechsler
Brian Seitra
Ray Jean
Application Number:
JP2020543237A
Publication Date:
November 15, 2023
Filing Date:
October 24, 2018
Export Citation:
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Assignee:
Sangamo Therapeutics, Incorporated
International Classes:
C12N15/62; A61K35/76; A61K38/46; A61K48/00; A61P21/02; A61P25/00; C07K14/435; C12N5/10; C12N15/09; C12N15/12; C12N15/55; C12N15/864
Domestic Patent References:
JP2016537341A
Foreign References:
WO2015153760A2
Other References:
Cell Mol Life Sci.,2007年,64(22),2933-2944
Attorney, Agent or Firm:
Norito Yamao
Norifumi Tomita
Fumio Inai
Manami Sasakura