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Related Concept Videos

Gene Therapy00:59

Gene Therapy

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Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be...
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Pharmacogenetics of Drug Targets: β₂-Adrenergic Receptors, Apo E, Thymidylate Synthase01:11

Pharmacogenetics of Drug Targets: β₂-Adrenergic Receptors, Apo E, Thymidylate Synthase

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Genetic polymorphisms in drug targets have emerged as critical determinants of interindividual variability in drug response and toxicity. Pharmacogenomic investigations increasingly focus on identifying these variations to personalize and optimize therapeutic interventions. A drug target may be a receptor, enzyme, or signaling protein involved in pharmacologic responses or disease-related pathways. While early pharmacogenetic studies focused primarily on drug metabolism, current research...
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iPS Cell Differentiation01:22

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The ability of induced pluripotent stem cells or iPSCs to differentiate into most body cell types has stimulated repair and regenerative medicine research over the past few decades. iPSC-derived blood cells, hepatocytes, beta islet cells, cardiomyocytes, neurons, and other cell types can repair injuries or regenerate damaged tissue in diseases such as diabetes and neurodegenerative disorders.
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Targeted Cancer Therapies02:57

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The targeted cancer therapies, also known as “molecular targeted therapies,” take advantage of the molecular and genetic differences between the cancer cells and the normal cells. It needs a thorough understanding of the cancer cells to develop drugs that can target specific molecular aspects that drive the growth, progression, and spread of cancer cells without affecting the growth and survival of other normal cells in the body.
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Bone Marrow Sampling and Transplants01:22

Bone Marrow Sampling and Transplants

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Bone marrow transplant is a potential cure for several diseases, including cancer and specific genetic disorders. Notably, this procedure is applicable for patients suffering from aplastic anemia, certain types of leukemia, severe combined immunodeficiency disease (SCID), Hodgkin's disease, non-Hodgkin's lymphoma, multiple myeloma, thalassemia, sickle-cell disease, and certain cancers.
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Related Experiment Video

Updated: Apr 24, 2026

Detection of Residual Donor Erythroid Progenitor Cells after Hematopoietic Stem Cell Transplantation for Patients with Hemoglobinopathies
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New Gene Therapy Strategy for β-Thalassemia.

Dongguo Liang1,2, Ingo G H Schmidt-Wolf3, Jingjing Pu4,5

  • 1Shanghai Institute of Hematology, National Research Center for Translational Medicine, State Key Laboratory of Medical Genomics, Ruijin Hospital, Shanghai Jiao Tong University School of Medicine, Shanghai, 20025, China.

Stem Cell Reviews and Reports
|April 23, 2026
PubMed
Summary

Gene therapy offers a promising new treatment for beta-thalassemia, a serious inherited blood disorder. Emerging gene editing techniques aim to restore normal hemoglobin production, moving towards a potential cure.

Keywords:
BCL11AFetal hemoglobin (HbF) reactivationGenome editingHemoglobin switchingβ-thalassemia

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Engineering Oncogenic Heterozygous Gain-of-Function Mutations in Human Hematopoietic Stem and Progenitor Cells
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Engineering Oncogenic Heterozygous Gain-of-Function Mutations in Human Hematopoietic Stem and Progenitor Cells
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Engineering Oncogenic Heterozygous Gain-of-Function Mutations in Human Hematopoietic Stem and Progenitor Cells

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Area of Science:

  • Hematology
  • Genetics
  • Molecular Biology

Background:

  • Beta-thalassemia is a prevalent inherited blood disorder causing chronic anemia and transfusion dependence.
  • Current curative options like stem cell transplantation have limitations in donor availability and complications.
  • Gene therapy has emerged as a transformative and promising alternative treatment.

Purpose of the Study:

  • To review established and emerging gene-based strategies for treating beta-thalassemia.
  • To discuss gene editing approaches targeting key regulatory elements for hemoglobin restoration.
  • To highlight advancements and challenges in gene therapy for beta-thalassemia.

Main Methods:

  • Review of lentiviral gene addition strategies to restore beta-globin (HBB) expression.
  • Analysis of gene editing techniques targeting BCL11A enhancer and HBG promoters.
  • Exploration of novel technologies like base editing and prime editing.

Main Results:

  • Gene therapy, including gene addition and editing, shows significant potential for beta-thalassemia treatment.
  • Targeting regulatory elements like the BCL11A enhancer can reactivate fetal hemoglobin (HBG).
  • Advanced gene editing tools offer precision and may reduce risks associated with DNA breaks.

Conclusions:

  • Gene therapy is rapidly advancing the treatment landscape for beta-thalassemia.
  • Key challenges include ensuring safety, durability, technical feasibility, and equitable access.
  • Gene therapy represents a significant step towards a broadly applicable curative approach for beta-thalassemia.