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Related Concept Videos

Gene Therapy00:59

Gene Therapy

27.6K
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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Cell Specific Gene Expression01:58

Cell Specific Gene Expression

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Multicellular organisms contain a variety of structurally and functionally distinct cell types, but the DNA in all the cells originated from the same parent cells. The differences in the cells can be attributed to the differential gene expression. Liver cells, whose functions include detoxification of blood, production of bile to metabolize fats, and synthesis of proteins essential for metabolism, must express a specific set of genes to perform their functions. Gene expression also varies with...
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Cell Specific Gene Expression

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Inflammatory Bowel Disease III: Diagnostic Studies and Management I-Nutritional Therapy01:30

Inflammatory Bowel Disease III: Diagnostic Studies and Management I-Nutritional Therapy

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Various diagnostic tests are employed in the diagnostic process for Inflammatory Bowel Disease (IBD), particularly to differentiate between Crohn's disease and ulcerative colitis.
Diagnostic studies
A colonoscopy is the definitive screening test, distinguishing ulcerative colitis from other colon diseases with similar symptoms. During a colonoscopy test, inflamed mucosa with exudate ulcerations can be observed, and biopsies are taken to determine the histologic characteristics of the...
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Stem Cell Therapy for Tissue Regeneration01:21

Stem Cell Therapy for Tissue Regeneration

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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.
Types of Stem Cells used in Stem Cell Therapy
The two main cell...
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Group Therapy01:26

Group Therapy

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Group therapy is a sociocultural approach to psychological treatment, where individuals with shared psychological challenges come together under the guidance of a mental health professional. This therapeutic modality offers unique opportunities for individuals to connect, share, and grow within the context of a supportive group. By fostering mutual understanding and collaboration, group therapy can address a range of psychological concerns effectively, often complementing or surpassing the...
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Related Experiment Video

Updated: Feb 2, 2026

Continuous Manual Exchange Transfusion for Patients with Sickle Cell Disease: An Efficient Method to Avoid Iron Overload
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Gene therapy for sickle cell disease.

Abiola Olowoyeye1, Charles I Okwundu

  • 1Children's Hospital Los Angeles, 1520 Rodney Drive apt 216, Los Angeles, California, USA.

The Cochrane Database of Systematic Reviews
|November 28, 2018
PubMed
Summary

Gene therapy shows promise for sickle cell disease (SCD), an inherited blood disorder. However, no clinical trials have been reported, highlighting the need for research to assess its safety and effectiveness.

Area of Science:

  • Hematology
  • Genetic Medicine
  • Gene Therapy

Background:

  • Sickle cell disease (SCD) is a group of genetic disorders caused by at least one hemoglobin S (Hb S) allele.
  • Autosomal recessive disorders like SCD are candidates for gene therapy due to the potential to restore normal function with a single gene copy.

Purpose of the Study:

  • To determine if gene therapy improves survival and prevents symptoms in sickle cell disease patients.
  • To evaluate the risks versus potential long-term benefits of gene therapy for SCD.

Main Methods:

  • Searched the Cochrane Haemoglobinopathies Trials Register, online registries, and relevant literature up to July 2018.
  • Included all randomized or quasi-randomized clinical trials (Phase 1-3) of gene therapy for sickle cell disease.

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Main Results:

  • No trials of gene therapy for sickle cell disease were identified.
  • No reported randomized or quasi-randomized clinical trials exist for gene therapy in SCD.

Conclusions:

  • Currently, no objective conclusions or recommendations can be made regarding gene therapy for sickle cell disease.
  • There is a critical need for well-designed, randomized controlled trials to assess the benefits and risks of gene therapy for SCD.