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Synthetic immunomodulation with a crispr super-repressor in vivo

An aptamer and amplicon technology applied in the field of synthetic immune regulation with CRISPR super-repressors in vivo

Pending Publication Date: 2021-08-31
ARIZONA STATE UNIVERSITY
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  • Summary
  • Abstract
  • Description
  • Claims
  • Application Information

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Problems solved by technology

Although extremely promising, the Cas9 protein from Streptococcus pyogenes (Sp-Cas9), the most common form of CRISPR studied to date, faces many challenges for clinical translation including potential immune response and genome specificity

Method used

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  • Synthetic immunomodulation with a crispr super-repressor in vivo
  • Synthetic immunomodulation with a crispr super-repressor in vivo
  • Synthetic immunomodulation with a crispr super-repressor in vivo

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example 1

[0091] The embodiments described herein demonstrate the use of a CRISPR-based synthetic repression system to repress endogenous MyD88 expression in vitro and in vivo.

[0092]Embodiments described herein employ previously reported concepts of CRISPR versatility (see Dahlman, J.E. et al. "Orthogonal gene knockout and activation with a catalytically active Cas9nuclease," Nature biotechnology 33, 1159 (2015) and Kiani, S. et al. "Cas9 gRNAengineering for genome editing, activation and repression," Nature methods 12, 1051 (2015), each of which is incorporated herein by reference). The ability to switch between nuclease-dependent and -independent functions of a single Cas9 protein offers the exciting possibility of combining gene editing with epigenetic manipulation. The inventors previously reported that truncating a guide RNA (gRNA) from the 5' end enabled the application of nuclease-competent Cas9 protein for transcriptional regulation of genes. Liao et al. recently demonstrate...

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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 Waldanstrom macroglobulinemia.

Description

[0001] Cross References to Related Applications [0002] This application claims priority to U.S. Provisional Application No. 62 / 757,679, filed November 8, 2018, and U.S. Provisional Application No. 62 / 899,584, filed September 12, 2019, each of which is incorporated in its entirety This article. [0003] Statement Regarding Federally Funded Research [0004] This invention was made with government support under R01 EB024562 awarded by the National Institutes of Health and HR0011-16-23657 awarded by DARPA. The government has certain rights in this invention. [0005] References to Sequence Listings Submitted via EFS-WEB [0006] The contents of the 48.4 kb ASCII text file of the Sequence Listing named "112624_01135_ST25.txt" created on November 7, 2019 and submitted electronically via EFS-Web are hereby incorporated by reference in their entirety. Background technique [0007] Adeno-associated viral (AAV) vectors hold broad potential for therapeutic gene delivery due t...

Claims

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Application Information

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Patent Type & Authority Applications(China)
IPC IPC(8): C07K14/315C12N9/22C12N15/63C12N15/90A61K48/00
CPCC12N15/90C12N9/22C07K2319/00C07K14/4703C12N15/635C12N2310/20C12N2750/14143C12N2830/005C12N15/113C12N2310/3519C12N2310/16A61K48/00A61P31/00A61K48/0008C12N15/111C12N15/86C07K2319/85C12N15/11
Inventor S·基亚尼M·R·易卜拉欣哈尼F·穆加达姆
Owner ARIZONA STATE UNIVERSITY
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