SgRNA editing HBB-41/42 deletion mutation site based on CRISPR/Cas9 technology

Abstract

An sgRNA editing 41/42 deletion mutation site of beta globin gene edit based on CRISPR/Cas9 technology is provided, and that nucleotide sequence thereof is shown as SEQ ID NO.1. The invention also provides a carrier associated therewith, a cell, a gene edit kit and application thereof, in particular a method for editing the 41/42 deletion mutation site of the beta globin gene based on the CRISPR/Cas9 technology, comprising constructing a CRISPR/Cas9 expression vector using sgRNA having a nucleotide sequence as shown in SEQ ID NO:1. The invention can specifically induce the traceless directional modification of gene mutation sites in beta globin gene 41/42 thalassemia, can selectively introduce single allele and double allele sequence changes with high efficiency and precision, and greatlypromotes the improvement of precise repair.

Description

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AI Technical Summary

Benefits of technology

This patented technology allows for precise correction or replacement of defect genes by introducing new modifications through specific DNA sequences introduced during cell culture processes. These modified versions have been found effective against certain types of inherited diseases such as sickle cell syndrome caused by beta hemoglobin deficiency.

Problems solved by technology

There have been identified various types of inherited diseases called sickle cell syndrome (SCD), including Sickness Hemolytic Disease (SSHD) and X Linked Labeled Disorders Type-1 , among others such as Cystic Fibrosis (CF)/Thyroid Glycoclonic Aciduria Syndromus Chloroencephalitis (THCA)-related neurodegenerative conditions like Spinal Cord Blood Cell Leukemias (SBLC)). These rare hereditary diseases cause life threatening complications during infancy when they affect young individuals who lack necessary healthcare facilities. Current methods involve either injecting replacement clonectomy mother kit containing prenatal serum aminotransferrin protein (pIg A) carrying specific sequences involved in iron metabolism pathways, or developing organoids derived from them. However these techniques require multiple generational cycles and may result in incomplete correction even if all relevant loci on chromatins are restored correctly.

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