iHSCs With the Gene Correction of HBB Intervent Subjests With β-thalassemia Mutations

General Information
Summary	This is a single centre, single arm, open-label study, to investigate the safety and efficacy of the gene correction of HBB in patient-specific iHSCs using CRISPR/Cas9.
Description	The purpose of this study is to evaluate the efficacy and safety of transplantation iHSCs intervent subjests with β-thalassemia mutations.
Clinical trials phase	Other
Start date (estimated)	2019-01-01
End date (estimated)	2021-01-31
Clinical feature	Label beta thalassemia Link http://purl.obolibrary.org/obo/DOID_12241 Description A thalassemia characterized by the reduced or absent synthesis of the beta globin chains of hemoglobin.; Xref MGI. OMIM mapping confirmed by DO. [SN].
Administrative Information
NCT number	NCT03728322
ICTRP weblink	https://trialsearch.who.int/Trial2.aspx?TrialID=NCT03728322
Other study identifiers	Name HBB HSC-01
Source weblink	https://clinicaltrials.gov/ct2/show/NCT03728322
Public contact	Email alife Public email allife@allifetech.com First name allife@allifetech.com City Beijing Country China
Sponsors	Allife Medical Science and Technology Co., Ltd.
Cells
Which differentiated cell type is used	Label hematopoietic stem cell Link http://purl.obolibrary.org/obo/CL_0000037 Description A stem cell from which all cells of the lymphoid and myeloid lineages develop, including blood cells and cells of the immune system. Hematopoietic stem cells lack cell markers of effector cells (lin-negative). Lin-negative is defined by lacking one or more of the following cell surface markers: CD2, CD3 epsilon, CD4, CD5 ,CD8 alpha chain, CD11b, CD14, CD19, CD20, CD56, ly6G, ter119.; Markers differ between species, and two sets of markers have been described for mice. HSCs are reportedly CD34-positive, CD45-positive, CD48-negative, CD150-positive, CD133-positive, and CD244-negative.
Recruitment
Recruitment Status	Not yet recruiting
Estimated number of participants	12