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Related Concept Videos

Gene Therapy00:59

Gene Therapy

26.4K
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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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.
The transplant begins with high doses of chemotherapy and radiation treatment, which aim to destroy...
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iPS Cell Differentiation01:22

iPS Cell Differentiation

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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 Duplication and Divergence02:37

Gene Duplication and Divergence

7.4K
The seminal work of Ohno in 1970 popularized the idea of gene duplication and divergence. DNA sequence comparison studies reveal that a large portion of the genes in bacteria, archaebacteria, and eukaryotes was  generated by gene duplication and divergence, indicating its critical role in evolution.
The duplicated copies of the gene are called Paralogs. Paralogs with similar sequences and functions form a gene family. Across several species, a large number of gene families are...
7.4K
Hemoglobin01:24

Hemoglobin

6.0K
Hemoglobin is a globular protein made up of four subunits. Two of these subunits are alpha chains, and the other two are beta chains. Each subunit contains a molecule of heme, which has an iron atom and can bind to oxygen. When an oxygen molecule binds to one heme group, it changes the shape of hemoglobin, making it easier for the other heme groups to bind oxygen as well.
When all four heme groups are bound to oxygen, the resulting molecule is called oxyhemoglobin. As a result, arterial blood...
6.0K
Gene Families01:57

Gene Families

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Gene families consist of groups of genes proposed to have originated from a common ancestor. Typically these arise through events in which a gene or genes are mistakenly duplicated during cell division. Unlike their parent genes (which are subject to selection pressure to maintain function), these gene copies do not need to preserve their sequences and may evolve at a relatively faster rate.
Occasionally these regions can be adapted to take on new roles within the organism, becoming novel genes...
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Engineering immune tolerance for AAV gene therapy: The Norse way.

Molecular therapy : the journal of the American Society of Gene Therapy·2026
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Hemophilia A gene therapy: Moving to the next chapter.

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Transient prophylactic immunosuppression with abatacept or dasatinib prevents immune responses in AAV gene transfer.

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Current Status of Clinical Gene Therapy for Hemophilia and Globin Disorders.

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Factor VIII originates primarily from anatomically distinct subsets of liver sinusoidal endothelial cells.

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Deaths in gene therapy of Duchenne muscular dystrophy and other diseases: Underlying mechanisms and mitigating strategies.

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Related Experiment Video

Updated: Nov 6, 2025

Preparation and Gene Modification of Nonhuman Primate Hematopoietic Stem and Progenitor Cells
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Preparation and Gene Modification of Nonhuman Primate Hematopoietic Stem and Progenitor Cells

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Old versus new gene therapy for globin disorders

Roland W Herzog1, Jörg Bungert2

  • 1Department of Pediatrics, Gene and Cell Therapy Program, Herman B. Wells Center for Pediatric Research, Indiana University School of Medicine, Indianapolis, IN 46202, USA.

Molecular Therapy : the Journal of the American Society of Gene Therapy
|May 7, 2021
PubMed
Summary

No abstract available in PubMed .

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