公开(公告)号：US12077771B2

公开(公告)日：2024-09-03

申请号：US18327373

申请日：2023-06-01

Applicant: ARIZONA BOARD OF REGENTS ON BEHALF OF ARIZONA STATE UNIVERSITY

Inventor： Samira Kiani ,  Mo Reza Ebrahimkhani ,  Jennifer Chapman

IPC: C12N15/85 ,  C12N9/22 ,  C12N15/11 ,  C12N15/115 ,  C12N15/62 ,  C12N15/90

CPC classification number: C12N15/85 ,  C12N9/22 ,  C12N15/11 ,  C12N15/115 ,  C12N15/62 ,  C12N15/907 ,  C12N2310/128 ,  C12N2310/16 ,  C12N2310/20 ,  C12N2501/12 ,  C12N2501/405 ,  C12N2800/80

Abstract: Aspects of the disclosure relate to methods and synthetic regulatory systems for more efficient nuclease-mediated homology-directed repair (HDR). In particular, provided herein are methods for more efficient in vivo and in vitro HDR-based gene editing where the methods comprise introducing into a cell a synthetic regulatory system comprising Cas nuclease, guide RNAs (gRNAs) having various lengths and configured to target distinct nucleotide sequences for simultaneous transcriptional repression (or activation) and genome editing via double stranded break and use of a donor nucleic acid molecule as a template for repair.

公开(公告)号：US11884925B2

公开(公告)日：2024-01-30

申请号：US17292407

申请日：2019-11-07

Applicant: ARIZONA BOARD OF REGENTS ON BEHALF OF ARIZONA STATE UNIVERSITY

Inventor： Samira Kiani ,  Mo Reza Ebrahimkhani ,  Farzaneh Moghadam

IPC: C12N15/63 ,  C07K14/47 ,  C12N9/22 ,  C12N15/11 ,  C12N15/86 ,  A61K48/00

CPC classification number: C12N15/635 ,  A61K48/0008 ,  C07K14/4703 ,  C12N9/22 ,  C12N15/11 ,  C12N15/111 ,  C12N15/86 ,  C07K2319/85 ,  C12N2310/16 ,  C12N2310/20 ,  C12N2310/3519 ,  C12N2830/005 ,  C12N2310/20 ,  C12N2310/16

Abstract: Provided herein are CRISPR-based synthetic repression systems as well as methods and compositions using the synthetic repression systems to treat septicemia, an adverse immune response in a subject and Waldenström macroglobulinemia.

公开(公告)号：US20220162574A1

公开(公告)日：2022-05-26

申请号：US17546810

申请日：2021-12-09

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

Inventor： Samira Kiani ,  Radwa Ewaisha ,  Shayesteh Roshdi Ferdosi ,  Karen Anderson ,  Farzaneh Moghadam ,  Sri Krishna ,  Mohammad R Ebrahimkhani

IPC: C12N9/22 ,  G01N33/573

Abstract: Provided herein are methods and compositions for reducing an undesirable T cell immune response in human patients prior to and/or during gene therapy using CRISPR/Cas9-based genetic modulation.

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.