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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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RNA editing is a post-transcriptional modification where a precursor mRNA (pre-mRNA) nucleotide sequence is changed by base insertion, deletion, or modification. The extent of RNA editing varies from a few hundred bases, in mitochondrial DNA of trypanosomes, to a just single base, in nuclear genes of mammals. Even a single base change in the pre-mRNA can convert a codon for one amino acid into the codon for another amino acid or a stop codon. This type of re-coding can significantly affect the...
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Tumor progression is a phenomenon where the pre-formed tumor acquires successive mutations to become clinically more aggressive and malignant. In the 1950s, Foulds first described the stepwise progression of cancer cells through successive stages.
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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.
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Hybridoma technology is used for the large-scale production of monoclonal antibodies. Monoclonal antibodies bind to only a single antigenic determinant or epitope. Such antibodies are used in research, diagnostics, and disease therapy. The hybridoma technology established in 1975 by Georges Köhler and Cesar Milstein was awarded the Nobel Prize in Medicine in 1984 for revolutionizing research and 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: Jan 22, 2026

CRISPR/Cas9 Gene Editing of Hematopoietic Stem and Progenitor Cells for Gene Therapy Applications
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[Gene editing technology and its recent progress in disease therapy].

Xu Ran Niu1, Shu Ming Yin1, Xi Chen1

  • 1Shanghai Key Laboratory of Regulatory Biology, School of Life Sciences, East China Normal University, Shanghai 200241, China.

Yi Chuan = Hereditas
|July 17, 2019
PubMed
Summary

Gene editing technology precisely modifies DNA/RNA for diverse applications. This review covers its development, strategies, and therapeutic potential for genetic diseases, including blood, liver, muscle, and nervous system disorders.

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Area of Science:

  • Biotechnology and Genetic Engineering
  • Molecular Biology
  • Therapeutic Development

Background:

  • Gene editing is a powerful genetic manipulation technology using bacterial nucleases for precise DNA/RNA modification.
  • It has established applications in research, breeding, and drug screening.
  • Increasing validity is seen in treating various diseases, particularly genetic disorders.

Purpose of the Study:

  • To review the evolution and diverse strategies of gene editing technology.
  • To explore current and potential applications of gene editing in disease therapy.
  • To summarize research on gene editing for genetic diseases affecting major organ systems.

Main Methods:

  • Review of scientific literature on gene editing technology development.
  • Analysis of gene editing strategies and their application in disease treatment.
  • Examination of research on gene editing therapies for blood, liver, muscle, and nervous system disorders.

Main Results:

  • Gene editing technologies offer accurate and efficient DNA/RNA modification.
  • Applications span basic research to therapeutic interventions for genetic diseases.
  • Specific research highlights base editing and epigenetic regulation for treating various systemic disorders.

Conclusions:

  • Gene editing therapy holds significant promise for treating a wide range of genetic diseases.
  • Continued research into base editors and epigenetic regulation will expand therapeutic options.
  • The future prospects for gene editing therapy are substantial, with ongoing development expected.