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Related Concept Videos

Gene Therapy00:59

Gene Therapy

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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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Microorganisms in Medicine and Therapeutics01:29

Microorganisms in Medicine and Therapeutics

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Microorganisms play a fundamental role in vaccine development, gene therapy, and therapeutic production. Their biological properties are harnessed to advance medicine and public health. Beyond immunization, microorganisms contribute to gut health, antibiotic synthesis, and genetic disease treatment.Live Attenuated and Inactivated VaccinesLive attenuated vaccines, such as the measles, mumps, and rubella (MMR) vaccine, utilize weakened forms of pathogens to closely resemble natural infections.
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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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Combination Therapies and Personalized Medicine02:50

Combination Therapies and Personalized Medicine

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Combining two or more treatment methods increases the life span of cancer patients while reducing damage to vital organs or tissue from the overuse of a single treatment. Combination therapy also targets different cancer-inducing pathways, thus reducing the chances of developing resistance to treatment.
The combination of the drug acetazolamide and sulforaphane is a good example of combination therapy to treat cancer. The cells in the interior of a large tumor often die due to the hypoxic and...
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Targeted Cancer Therapies02:57

Targeted Cancer Therapies

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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.
There are several types of targeted therapies against...
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Gene Therapy for Sickle Cell Disease: Practice Recommendations from the American Society for Transplantation and Cellular Therapy and the International Society for Cell & Gene Therapy.

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The role of the conditioning regimen for autologous and ex vivo genetically modified hematopoietic stem cell-based therapies: recommendations from the ISCT stem cell engineering committee.

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Spin device-based image edge detection architecture for neuromorphic computing.

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Gene Therapies for Transfusion-Dependent b-Thalassemia.

Indian pediatrics·2021
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CRISPR-Cas9 Gene Editing for Sickle Cell Disease and β-Thalassemia.

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Gene therapies for transfusion dependent β-thalassemia: Current status and critical criteria for success.

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Dialysis bioreactor enables high-density, serum-free expansion of dental pulp-derived mesenchymal stromal cells with enhanced secretome.

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Beyond the synapses: should we anticipate neurotransmitter signaling when manufacturing CAR-T cells for brain tumors?

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Curative treatment for severe sickle cell disease: allogeneic hematopoietic cell transplant or gene therapy.

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Manufacturing strategies for prolonged CAR-T cell persistence.

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Overcoming five main challenges to targeting hematologic malignancies.

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Related Experiment Video

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Author Spotlight: Addressing Regulatory Gaps in Molecular Studies by Quantifying Viral Vectors in Complex Matrices
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2023 Gene Therapy Mini-Series

Sandeep Soni1

  • 1Pediatrics, University of California, San Francisco, San Francisco, California, USA; Crispr Therapeutics AG, Boston, Massachusetts, USA; ISCT Immune-Gene Therapy Committee, ISCT, Vancouver, California, USA.

Cytotherapy
|February 1, 2023
PubMed
Summary

No abstract available in PubMed .

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