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Related Concept Videos

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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Gene Therapy00:59

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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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Stem cell therapy is a method used in regenerative medicine to repair and restore function to damaged tissues and organs. Stem cells have the potential to proliferate and differentiate into various tissue types, making them ideal candidates for tissue regeneration. For example, hematopoietic stem cell transplants are commonly used in blood cancer treatment to replenish damaged bone marrow and restore healthy blood cells.
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Bone Marrow Sampling and Transplants01:22

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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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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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X-linked Traits01:19

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In most mammalian species, females have two X sex chromosomes and males have an X and Y. As a result, mutations on the X chromosome in females may be masked by the presence of a normal allele on the second X. In contrast, a mutation on the X chromosome in males more often causes observable biological defects, as there is no normal X to compensate. Trait variations arising from mutations on the X chromosome are called “X-linked”.
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Phosphorylated vasodilator-stimulated phosphoprotein (P-VASPSer239) in platelets is increased by nitrite and partially deoxygenated erythrocytes.

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Related Experiment Video

Updated: Jun 4, 2025

A Precision Medicine Tool for Measurement and Monitoring of Hemoglobin S in Sickle Cell Disease Patients Receiving Transfusion Therapy
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Sickle cell anaemia therapy in 2025.

Alan N Schechter1

  • 1Molecular Medicine Branch, National Institute of Diabetes and Digestive and Kidney Diseases, National Institutes of Health, Bethesda, Maryland, USA.

British Journal of Haematology
|December 23, 2024
PubMed
Summary
This summary is machine-generated.

New sickle cell anaemia treatments show limited success. This review questions if hydroxyurea, a current therapy, deserves more focus given the slow progress of stem cell and gene therapies.

Keywords:
fetal haemoglobinhydroxyureasickle cell anaemiatherapy

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Area of Science:

  • Hematology
  • Pharmacology
  • Genetic Medicine

Background:

  • Sickle cell anaemia (SCA) management faces challenges with novel therapies.
  • Curative stem cell and gene therapies are not yet widely applicable for SCA.

Purpose of the Study:

  • To evaluate the current role and potential of hydroxyurea in sickle cell anaemia management.
  • To address the need for effective and accessible SCA treatments.

Main Methods:

  • Review of existing literature on SCA pharmacotherapies.
  • Analysis of the efficacy and accessibility of hydroxyurea compared to emerging treatments.

Main Results:

  • Recent pharmacological therapies for SCA have not met expectations.
  • Widespread implementation of stem cell and gene therapies for SCA is not imminent.

Conclusions:

  • Hydroxyurea remains a critical therapeutic option for sickle cell anaemia.
  • Further investigation into optimizing hydroxyurea use is warranted given current treatment limitations.