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Related Concept Videos

iPS Cell Differentiation01:22

iPS Cell Differentiation

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.
Gene Therapy00:59

Gene Therapy

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 inserted. The...
Bone Marrow Sampling and Transplants01:22

Bone Marrow Sampling and Transplants

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.
The transplant begins with high doses of chemotherapy and radiation treatment, which aim to destroy the...
Multiple Allele Traits01:49

Multiple Allele Traits

The Concept of Multiple Allelism
EPS and iPS Cells in Disease Research01:21

EPS and iPS Cells in Disease Research

Embryonic and induced pluripotent stem cells are excellent models for disease research because of their ability to self-renew and differentiate into most cell types. Somatic cells from a patient are isolated and reprogrammed into induced pluripotent stem cells or iPSCs. These iPSCs are later differentiated into the desired cell type, which mirrors the diseased cell of the patient. In this way, disease models have been created for investigating diseases such as Down syndrome, type I diabetes,...
Regulation of Hematopoietic Stem Cells01:01

Regulation of Hematopoietic Stem Cells

All blood and immune cells are produced from the multipotent hematopoietic stem cells (HSCs) by the process of hematopoiesis. However, they all have a limited life span. In addition, many are depleted in immune surveillance or combatting an injury or infection. This makes blood one of the most regenerative tissues. Hematopoiesis helps replenish these blood and immune cells, restoring the body's normal functioning. However, overproduction of blood and immune cells can make them cancerous or...

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Related Experiment Video

Updated: Jun 12, 2026

Characterization of Sickling During Controlled Automated Deoxygenation with Oxygen Gradient Ektacytometry
08:23

Characterization of Sickling During Controlled Automated Deoxygenation with Oxygen Gradient Ektacytometry

Published on: November 5, 2019

Sickle Cell Disease: Historical Overview and Current Therapies.

Oluwaseun O Babatunde1, Madeleine G Bibby1, Anthony Atala1

  • 1Wake Forest Institute for Regenerative Medicine, Winston-Salem, North Carolina, USA.

Prenatal Diagnosis
|June 11, 2026
PubMed
Summary

Sickle cell disease (SCD) affects millions globally. In utero transplantation (IUTx) offers a potential cure before birth, avoiding current treatment limitations and risks.

Keywords:
HSC transplantationclinical managementhematopoietic stem cell (HSC)pathophysiologysickle cell disease

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Continuous Manual Exchange Transfusion for Patients with Sickle Cell Disease: An Efficient Method to Avoid Iron Overload
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Continuous Manual Exchange Transfusion for Patients with Sickle Cell Disease: An Efficient Method to Avoid Iron Overload

Published on: March 14, 2017

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Last Updated: Jun 12, 2026

Characterization of Sickling During Controlled Automated Deoxygenation with Oxygen Gradient Ektacytometry
08:23

Characterization of Sickling During Controlled Automated Deoxygenation with Oxygen Gradient Ektacytometry

Published on: November 5, 2019

Continuous Manual Exchange Transfusion for Patients with Sickle Cell Disease: An Efficient Method to Avoid Iron Overload
05:23

Continuous Manual Exchange Transfusion for Patients with Sickle Cell Disease: An Efficient Method to Avoid Iron Overload

Published on: March 14, 2017

Area of Science:

  • Hematology
  • Genetics
  • Pediatric Medicine

Background:

  • Sickle cell disease (SCD) is a prevalent genetic disorder with limited therapeutic options.
  • Current treatments, including hematopoietic stem cell (HSC) transplantation, have significant risks and side effects.
  • SCD manifestations begin in infancy, posing a risk of irreversible organ damage.

Purpose of the Study:

  • To review the history and current limitations of SCD treatments.
  • To introduce in utero transplantation (IUTx) as a potential curative therapy for SCD.
  • To highlight the benefits of treating SCD before birth.

Main Methods:

  • Literature review of SCD history and treatments.
  • Conceptual overview of in utero transplantation (IUTx) for SCD.
  • Analysis of current therapeutic challenges and unmet needs.

Main Results:

  • Existing SCD treatments are insufficient and carry substantial risks.
  • Hematopoietic stem cell (HSC) transplantation is curative but challenging.
  • In utero transplantation (IUTx) presents a novel approach to correct SCD prenatally.

Conclusions:

  • There is an urgent need for novel, curative SCD therapies.
  • In utero transplantation (IUTx) could prevent disease onset and lifelong complications.
  • Prenatal treatment of SCD offers a promising alternative to postnatal interventions.