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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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To learn more about the function of a gene, researchers can observe what happens when the gene is inactivated or “knocked out,” by creating genetically engineered knockout animals. Knockout mice have been particularly useful as models for human diseases such as cancer, Parkinson’s disease, and diabetes.
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Genome editing technologies allow scientists to modify an organism’s DNA via the addition, removal, or rearrangement of genetic material at specific genomic locations. These types of techniques could potentially be used to cure genetic disorders such as hemophilia and sickle cell anemia. One popular and widely used DNA-editing research tool that could lead to safe and effective cures for genetic disorders is the CRISPR-Cas9 system. CRISPR-Cas9 stands for Clustered Regularly Interspaced...
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Transdifferentiation, also known as lineage reprogramming, was first discovered by Selman and Kafatos in 1974 in silkmoths. They observed that the moths’ cuticle-producing cells transformed into salt-producing cells. Many such cases of natural transdifferentiation occur in organisms. In humans, pancreatic alpha cells can become beta cells. In newts, the loss of the eye’s lens causes the pigmented epithelial cells to transdifferentiate into the lens cells.
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Cancer therapies are various modes of treatment, such as surgery, radiation therapy, and chemotherapy that are administered to cancer patients.
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Demystifying gene(tic) therapies.

Chun-Hung Chan1, David A Pearce2

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Summary
This summary is machine-generated.

This article highlights resources for understanding gene therapies. It addresses the need for better education for researchers, clinicians, patients, and policymakers on genetic therapeutic approaches.

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Area of Science:

  • Biomedical Sciences
  • Genetics
  • Therapeutics

Background:

  • The field of gene therapy is rapidly evolving, presenting challenges in education for diverse stakeholders.
  • A session at the RE(ACT) congress and IRDiRC conference focused on demystifying gene therapies.
  • Existing educational resources may not adequately address the complexities of genetic therapeutic approaches.

Purpose of the Study:

  • To summarize discussions on the evolving landscape of gene therapies.
  • To identify and highlight resources that can aid in understanding various genetic therapeutic approaches.
  • To address the educational needs of stakeholders involved in gene therapy.

Main Methods:

  • Summary of a scientific session discussion.
  • Identification of relevant educational resources.
  • Focus on stakeholder education in gene therapy.

Main Results:

  • Gene therapy education requires enhanced resources for researchers, clinicians, patients, and advocates.
  • A variety of genetic therapeutic approaches exist, necessitating clear explanations.
  • The article aims to demystify complex genetic therapies by pointing to useful resources.

Conclusions:

  • Improved educational strategies and resources are crucial for the advancement and adoption of gene therapies.
  • Demystifying gene therapies is essential for informed decision-making by all stakeholders.
  • Continued dialogue and resource sharing are vital for navigating the complexities of genetic medicine.