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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.
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Multiple Allele Traits

The Concept of Multiple Allelism
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...
Cystic Fibrosis: Management01:24

Cystic Fibrosis: Management

Cystic fibrosis (CF) is an autosomal recessive disorder that predominantly affects individuals of Northern European descent, occurring at a rate of 1 in 3500. It is caused by a genetic mutation in a gene on chromosome 7, most commonly the ΔF508 mutation, that codes for the cystic fibrosis transmembrane conductance regulator (CFTR) protein. This results in thicker mucus secretions and obstruction pathologies in multiple organs, including the lungs and sinuses.
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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...
Pharmacogenomics: Identification of New Drug Targets01:29

Pharmacogenomics: Identification of New Drug Targets

Advances in genomics have profoundly influenced drug discovery by increasing both the speed and accuracy of pharmaceutical development. Pharmacogenomics, which examines how genetic variation influences drug response, facilitates the identification of novel therapeutic targets and enables patient stratification for personalized treatment. These strategies contribute to improved drug efficacy, minimized adverse effects, and more efficient clinical trial design.Mapping genetic differences...

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Related Experiment Video

Updated: Jun 23, 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

[Advances in sickle cell disease].

Mariane de Montalembert1

  • 1Pédiatrie générale, Hôpital Necker-Enfants Malades, Paris. mariane.demontal@nck.aphp.fr

Bulletin De L'Academie Nationale De Medecine
|May 19, 2009
PubMed
Summary
This summary is machine-generated.

New research in sickle cell disease reveals underlying mechanisms like inflammation and vascular issues. Current treatments focus on red blood cell health and fetal hemoglobin, with bone marrow transplant and gene therapy offering cures.

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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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Last Updated: Jun 23, 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
  • Immunology

Context:

  • Sickle cell disease pathophysiology involves a persistent proinflammatory state and vascular tone dysregulation.
  • Transgenic mouse models have elucidated novel disease mechanisms.
  • Therapeutic strategies are evolving beyond symptomatic relief.

Purpose:

  • To review recent advancements in understanding sickle cell disease mechanisms.
  • To highlight emerging therapeutic targets and treatments.
  • To discuss the potential of bone marrow transplantation and gene therapy.

Summary:

  • Novel insights from transgenic mice reveal chronic inflammation and altered vascular tone in sickle cell disease.
  • Emerging treatments target red blood cell hydration and deoxyhemoglobin S polymerization kinetics.
  • Hydroxyurea is a key therapy, while anti-adhesion molecules and vascular modulating agents are under investigation.

Impact:

  • Advances in understanding sickle cell disease are driving the development of targeted therapies.
  • Bone marrow transplantation offers a cure for eligible patients.
  • Gene therapy presents a promising future treatment for individuals lacking a suitable donor.