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Related Concept Videos

Gene Therapy00:59

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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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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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Genetic screens are tools used to identify genes and mutations responsible for phenotypes of interest. Genetic screens help identify individuals or a group of people at risk of developing  genetic diseases and help them with early intervention, targeted therapy, and reproductive options.
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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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Generation of Defined Genomic Modifications Using CRISPR-CAS9 in Human Pluripotent Stem Cells
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Gene surgery: Potential applications for human diseases.

Ayman El-Kenawy1,2, Bachir Benarba3, Adriana Freitas Neves4

  • 1Department of Pathology, College of Medicine, Taif University, Saudi Arabia.

EXCLI Journal
|November 26, 2019
PubMed
Summary
This summary is machine-generated.

Gene surgery, particularly using CRISPR-Cas, offers promising therapeutic applications for diseases like cancer and diabetes. However, challenges in design, delivery, and ethical considerations remain for this emerging gene editing technology.

Keywords:
CRISPR-Cascancerdiabetesgene surgeryobesity

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Area of Science:

  • Biotechnology
  • Genetics
  • Medical Science

Background:

  • Gene therapy has emerged as a significant therapeutic approach for various diseases.
  • CRISPR-Cas technology has advanced gene editing capabilities for eukaryotic cells.

Purpose of the Study:

  • To review the applications of gene surgery in treating human diseases.
  • To discuss the molecular mechanisms underlying gene surgery's therapeutic effects.
  • To highlight the role of CRISPR-Cas in gene surgery.

Main Methods:

  • Review of existing studies on gene surgery applications.
  • Analysis of molecular mechanisms in gene therapy.
  • Focus on CRISPR-Cas, ZFNs, and TALENs technologies.

Main Results:

  • Gene surgery shows therapeutic potential in cancer, diabetes (Type 1), cardiovascular diseases, neurological disorders, and β-thalassemia.
  • CRISPR-Cas is effective in the diagnosis, screening, and treatment of various cancers.
  • Gene surgery effectively targets signaling pathways in Type 1 diabetes.

Conclusions:

  • Gene surgery, especially with CRISPR-Cas, presents significant therapeutic potential across multiple human diseases.
  • Limitations include design complexities, cost, off-target effects, delivery challenges, and ethical concerns.
  • Further research is needed to overcome these limitations for widespread clinical application.