Compositions and methods for treating hemoglobin diseases
CN122003501APending Publication Date: 2026-05-08SHANGHAI VITALGEN BIOPHARMA CO LTD
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Patent Information
- Application Number
- CN202480062400.7
- Authority / Receiving Office
- CN · China
- Patent Type
- Applications(China)
- Current Assignee / Owner
- Priority Date
- 2023-09-28
- Filing Date
- 2024-09-27
- Publication Date
- 2026-05-08
AI Technical Summary
Technical Problem
The prior art has limitations in the treatment of hemoglobin diseases, especially in the difficulty of providing safe and effective treatments, and requires a fully matched bone marrow or cord blood donor to avoid the risk of post-transplant GVHD.
Method used
Increase the level of HbF in human erythrocyte progeny of human CD34+HSPCs by genome editing, and targeted editing in the transcriptional regulatory regions of HBG1 and HBG2 using guide RNA (gRNA) and CRISPR/Cas systems to treat β-thalassemia and sickle cell disease.
Benefits of technology
This method can significantly increase the expression level of HbF, reduce dependence on fully matched donors, reduce the risk of GVHD after transplantation, and provide a potential cure for beta-hemoglobinosis.
✦ Generated by Eureka AI based on patent content.
Abstract
Provided are gRNA molecules, and nucleic acids, complexes, compositions, cells, and kits thereof. Also provided are methods of increasing the expression level of HbF in progeny erythrocytes of human CD34 + HSPCs by genome editing, which are useful in the treatment of hemoglobinopathies, such as beta-thalassemia and sickle cell disease.
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