公开(公告)号：US11946046B2

公开(公告)日：2024-04-02

申请号：US15734174

申请日：2019-06-14

Applicant: University of Utah Research Foundation

Inventor： Stefan M. Pulst ,  Daniel R. Scoles ,  Sharan Paul

IPC: C12N15/113 ,  A61K45/06

CPC classification number: C12N15/113 ,  A61K45/06 ,  C12N2310/11 ,  C12N2310/12 ,  C12N2310/141 ,  C12N2320/32

Abstract: Methods of minimizing dysregulation of Staufen1-associated RNA metabolism can include introducing an amount of a Staufen1-regulating agent to a target cell sufficient to minimize the dysregulation. Therapeutic compositions for treating a neurodegenerative condition associated with Staufen1-induced dysregulation of RNA metabolism can include a therapeutically effective amount of a Staufen1-regulating agent and a pharmaceutically acceptable carrier.

公开(公告)号：US20190017047A1

公开(公告)日：2019-01-17

申请号：US15973088

申请日：2018-05-07

Applicant: Ionis Pharmaceuticals, Inc. ,  University of Utah Research Foundation

Inventor： C. Frank Bennett ,  Susan M. Freier ,  Stefan M. Pulst ,  Daniel R. Scoles ,  Gene Hung

IPC: C12N15/113

CPC classification number: C12N15/113 ,  C12N2310/11 ,  C12N2310/14 ,  C12N2310/141 ,  C12N2310/315 ,  C12N2310/317 ,  C12N2310/321 ,  C12N2310/323 ,  C12N2310/3231 ,  C12N2310/3341 ,  C12N2310/341 ,  C12N2310/346 ,  C12N2310/3525

Abstract: Disclosed herein are methods for decreasing Ataxin 2 mRNA and protein expression. Such methods are useful to treat, prevent, or ameliorate Ataxin 2 associated diseases, disorders, and conditions. Such Ataxin 2 associated diseases include spinocerebellar ataxia type 2 (SCA2), amyotrophic lateral sclerosis (ALS), and parkinsonism.

公开(公告)号：US09920333B2

公开(公告)日：2018-03-20

申请号：US14984861

申请日：2015-12-30

Applicant: University of Utah Research Foundation

Inventor： Stefan M. Pulst ,  Sharan Paul ,  Warunee Dansithong

IPC: C12N5/00 ,  C12N15/86 ,  C12N5/074 ,  C12N5/0793 ,  C12N15/85

CPC classification number: C12N15/86 ,  C12N5/0619 ,  C12N5/0696 ,  C12N15/85 ,  C12N2501/602 ,  C12N2501/603 ,  C12N2501/604 ,  C12N2501/606 ,  C12N2510/00 ,  C12N2710/10043 ,  C12N2710/10343

Abstract: Systems, constructs, and methods for reprogramming cells are provided. In one aspect, for example, a transformation construct for generating iPS cells can include an expression vector having a plurality of reprogramming factors, each reprogramming factor being under control of a separate promoter.

公开(公告)号：US09228204B2

公开(公告)日：2016-01-05

申请号：US13975004

申请日：2013-08-23

Applicant: University of Utah Research Foundation

Inventor： Stefan M. Pulst ,  Sharan Paul ,  Warunee Dansithong

IPC: C12N15/00 ,  C12N15/86 ,  C12N5/074

CPC classification number: C12N15/86 ,  C12N5/0619 ,  C12N5/0696 ,  C12N15/85 ,  C12N2501/602 ,  C12N2501/603 ,  C12N2501/604 ,  C12N2501/606 ,  C12N2510/00 ,  C12N2710/10043 ,  C12N2710/10343

Abstract: Systems, constructs, and methods for reprogramming cells are provided. In one aspect, for example, a transformation construct for generating iPS cells can include an expression vector having a plurality of reprogramming factors, each reprogramming factor being under control of a separate promoter.

Abstract translation: 提供了用于重新编程细胞的系统，构建体和方法。 在一个方面，例如，用于产生iPS细胞的转化构建体可以包括具有多个重编程因子的表达载体，每个重编程因子都由单独的启动子控制。