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

2018-11-07 17:13:11 | BioPortfolio


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.


The purpose of this study is to evaluate the efficacy and safety of transplantation iHSCs intervent subjests with β-thalassemia mutations.

Study Design




iHSCs treatment group


Not yet recruiting


Allife Medical Science and Technology Co., Ltd.

Results (where available)

View Results


Published on BioPortfolio: 2018-11-07T17:13:11-0500

Clinical Trials [308 Associated Clinical Trials listed on BioPortfolio]

Complications in Children With B- Thalassemia Major

Worldwide, there are more than 60,000 births annually of serious forms of thalassemia .The World Health Organization considers thalassemia to be a major health burden. Beta- thalassemia i...

Evaluating People With Thalassemia: The Thalassemia Longitudinal Cohort (TLC) Study

Thalassemias are inherited blood disorders that can cause anemia and other health problems. The goal of this study is to collect information on complications of the disease among people wh...

Assessment of Pain in People With Thalassemia

Thalassemia is an inherited blood disorder that can result in mild to severe anemia. People with thalassemia often experience pain, but the exact sources and prevalence of pain remain unkn...

Zinc to Improve Bone Health in People With Thalassemia (The Think Zinc Study)

Many people with the blood disorder thalassemia also have osteoporosis, possibly caused by low zinc levels. This study will evaluate the effectiveness of zinc supplements at improving bone...

Assessment of Pain in People With Thalassemia Who Are Treated With Regular Blood Transfusions

Thalassemia is an inherited blood disorder that can result in mild to severe anemia. Regular blood transfusions, which refresh the healthy red blood cell supply, are one treatment for thal...

PubMed Articles [23597 Associated PubMed Articles listed on BioPortfolio]

Misdiagnosis of double heterozygous εγ(γδβ)-thalassemia/β thalassemia as homozygous β-thalassemia: A pitfall for molecular diagnostic laboratories.

Compound heterozygosity of a silent beta-thalassemia mutation at the 3'-untranslated region (HBB: c.*132 C>T) and beta-zero thalassemia results in thalassemia intermedia.

Impact of the detection of ζ-globin chains and hemoglobin Bart's using immunochromatographic strip tests for α0-thalassemia (--SEA) differential diagnosis.

α0-Thalassemia is an inherited hematological disorder caused by the deletion of α-globin genes. The Southeast Asian deletion (--SEA) is the most common type of α0-thalassemia observed in Southeast ...

Discrimination of β-thalassemia and iron deficiency anemia through extreme learning machine and regularized extreme learning machine based decision support system.

The symptoms of Iron Deficiency Anemia (IDA) and β-thalassemia (β-TT) disease are similar and the distinction between them is time consuming and costly. There are several indices used to differentia...

Management of age-associated medical complications in patients with β-thalassemia.

: β-Thalassemia syndromes are among the most common monogenic disorders worldwide. Clinically, on the basis of the severity of the phenotype, β-thalassemias are classified into two groups: transfusi...

Medical and Biotech [MESH] Definitions

A disorder characterized by reduced synthesis of the beta chains of hemoglobin. There is retardation of hemoglobin A synthesis in the heterozygous form (thalassemia minor), which is asymptomatic, while in the homozygous form (thalassemia major, Cooley's anemia, Mediterranean anemia, erythroblastic anemia), which can result in severe complications and even death, hemoglobin A synthesis is absent.

A hereditary disorder characterized by reduced or absent DELTA-GLOBIN thus effecting the level of HEMOGLOBIN A2, a minor component of adult hemoglobin monitored in the diagnosis of BETA-THALASSEMIA.

A group of hereditary hemolytic anemias in which there is decreased synthesis of one or more hemoglobin polypeptide chains. There are several genetic types with clinical pictures ranging from barely detectable hematologic abnormality to severe and fatal anemia.

Withholding or withdrawal of a particular treatment or treatments, often (but not necessarily) life-prolonging treatment, from a patient or from a research subject as part of a research protocol. The concept is differentiated from REFUSAL TO TREAT, where the emphasis is on the health professional's or health facility's refusal to treat a patient or group of patients when the patient or the patient's representative requests treatment. Withholding of life-prolonging treatment is usually indexed only with EUTHANASIA, PASSIVE, unless the distinction between withholding and withdrawing treatment, or the issue of withholding palliative rather than curative treatment, is discussed.

Refusal of the health professional to initiate or continue treatment of a patient or group of patients. The refusal can be based on any reason. The concept is differentiated from PATIENT REFUSAL OF TREATMENT see TREATMENT REFUSAL which originates with the patient and not the health professional.

More From BioPortfolio on "iHSCs With the Gene Correction of HBB Intervent Subjests With β-thalassemia Mutations"

Quick Search

Relevant Topics

Bioinformatics is the application of computer software and hardware to the management of biological data to create useful information. Computers are used to gather, store, analyze and integrate biological and genetic information which can then be applied...

Organ transplantation is the moving of an organ from one body to another or from a donor site to another location on the patient's own body, for the purpose of replacing the recipient's damaged or absent organ. The emerging field of regenerative ...

Searches Linking to this Trial