公开(公告)号：US11773407B2

公开(公告)日：2023-10-03

申请号：US16626021

申请日：2018-06-26

Applicant: Arizona Board of Regents on behalf of Arizona State University

Inventor： Samira Kiani ,  Mo Reza Ebrahimkhani ,  Swechchha Pradhan ,  Farzaneh Moghadam

IPC: C12N15/86 ,  C12N9/22 ,  C12N15/113

CPC classification number: C12N15/86 ,  C12N9/22 ,  C12N15/113 ,  C12N2310/20 ,  C12N2750/14143

Abstract: Aspects of the disclosure relate to synthetic regulatory systems composed of a multifunctional Cas nuclease, at least two guide RNAs (gRNAs) configured to target distinct nucleotide sequences, and a multilayered regulatory control element comprising ribozyme-based safety switches providing spatial and temporal control over the synthetic circuit in vivo.

公开(公告)号：US20210154326A1

公开(公告)日：2021-05-27

申请号：US16626013

申请日：2018-06-26

Applicant: Arizona Board of Regents on behalf of Arizona State University

Inventor： Samira Kiani ,  Swechchha Pradhan ,  Farzaneh Moghadam ,  Mo Reza Ebrahimkhani

IPC: A61K48/00 ,  C12N15/113 ,  C12N15/86 ,  C12N9/22

Abstract: Aspects of the disclosure relate to synthetic regulatory systems comprising a multifunctional Cas nuclease and at least two guide RNAs (gRNAs) including a truncated gRNA and an multilayered regulatory control element. The synthetic regulatory system modulates endogenous gene expression, including transcriptional repression and transcriptional activation of one or more endogenous genes of a mammalian inner ear cell with multiple safety switches. Also provided herein are methods for modulating hearing sensitivity in damaged cells of the organ of corti.

公开(公告)号：US20210155954A1

公开(公告)日：2021-05-27

申请号：US16626021

申请日：2018-06-26

Applicant: Arizona Board of Regents on Behalf of Arizona State University

Inventor： Samira Kiani ,  Mo Reza Ebrahimkhani ,  Swechchha Pradhan ,  Farzaneh Moghadam

IPC: C12N15/86 ,  C12N9/22 ,  C12N15/113

Abstract: Aspects of the disclosure relate to synthetic regulatory systems composed of a multifunctional Cas nuclease, at least two guide RNAs (gRNAs) configured to target distinct nucleotide sequences, and a multilayered regulatory control element comprising ribozyme-based safety switches providing spatial and temporal control over the synthetic circuit in vivo.