GRNA molecule targeting beta-globin gene, synthetic method thereof and method for correcting mutation type

A globin and molecular technology, applied in the field of correcting the type of β-globin gene mutation in β-thalassemia and repairing the system, can solve problems such as limitations in promotion and use, and achieve no off-target mutations, efficient targeted insertion, and small mutations. effect of load

Pending Publication Date: 2021-09-24
SUSHENG BIOTECH (HAINAN) CO LTD
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  • Abstract
  • Description
  • Claims
  • Application Information

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Problems solved by technology

However, the generalization of this approach has been limited by the lack of immunologically matched donors and graft-versus-host disease [4]

Method used

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  • GRNA molecule targeting beta-globin gene, synthetic method thereof and method for correcting mutation type
  • GRNA molecule targeting beta-globin gene, synthetic method thereof and method for correcting mutation type
  • GRNA molecule targeting beta-globin gene, synthetic method thereof and method for correcting mutation type

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Embodiment Construction

[0045] Exemplary embodiments of the present application are described below with reference to the accompanying drawings, including various details of the embodiments of the present application to facilitate understanding, and they should be considered as exemplary only. Accordingly, those of ordinary skill in the art will recognize that various changes and modifications of the embodiments described herein can be made without departing from the scope and spirit of the application. Similarly, descriptions of well-known functions and constructions are omitted in the following description for clarity and conciseness.

[0046] It should be noted that all methods and processes not described in detail in the specification use steps and operations known in the art, and thus will not be repeated herein.

[0047] The abbreviated terms used in the examples herein mainly include: HSPC: hematopoietic stem cells and progenitor cells; CRISPR / Cas: clustered regularly interspaced short palindr...

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Abstract

The invention discloses a gRNA molecule of an intron I or an intron II targeting beta-globin gene, a synthesis method of the gRNA molecule, a method for constructing a repair system of a targeted beta-globin gene mutation site, the repair system and a universal method for correcting a beta-globin gene mutation type in beta-thalassemia. Particularly, the general method can correct most types of HBB gene mutations in HSPC, and the targeted HSPC has a normal function in vitro and does not have an obvious off-target effect.

Description

technical field [0001] This application relates to the field of biotechnology, in particular to a gRNA molecule targeting intron I or intron II of the β-globin gene, its synthesis method, and a gRNA molecule targeting the mutation site of the β-globin gene A method of repairing the system, a repairing system, and a general method for correcting the mutation type of the β-globin gene in β-thalassemia. Background technique [0002] It is well known that β-thalassemia (abbreviated as β-thalassemia) is caused by more than 200 different types of mutations in the β-globin (HBB) gene [1]. Normally, HBB pairs one-to-one with alpha-globulin (HBA) to form a tetrameric hemoglobin molecule, and due to insufficient production of HBB, unpaired alpha-globin chains precipitate, resulting in developing erythrocytes or erythroid precursors Toxic death occurs with insufficient formation of mature red blood cells (RBCs) [2]. Failed erythropoiesis leads to anemia, and if left untreated, severe...

Claims

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Application Information

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Patent Type & Authority Applications(China)
IPC IPC(8): C12N15/113C12N15/10C12N15/864C12N15/90
CPCC12N15/113C12N15/10C12N15/86C12N15/907C07K14/805C12N2310/20C12N2750/14143C12N2800/107C12Q2531/113C12N9/22A61K48/005A61K31/7105
Inventor 曾胜陈鑫苹杨宗繁丰玫玫王烨
Owner SUSHENG BIOTECH (HAINAN) CO LTD
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