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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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Introduction to Nuclear Reprogramming01:14

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Nuclear reprogramming is the process of switching gene expression of one cell type to that of another cell type, usually from a differentiated cell state to an undifferentiated cell state. Differentiation occurs during processes such as development and morphogenesis, tissue regeneration, and malignancy. Cells can also be artificially induced to reprogram their gene expression by techniques such as nuclear transfer, induced pluripotency, and cell fusion. Such techniques have many applications in...
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Methods of Nuclear Reprogramming01:24

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Nuclear reprogramming is a process of transforming one cell type into an unrelated cell type by epigenetic changes that alter the cell’s original gene expression pattern. Such epigenetic changes force cells to express a different set of genes, which play a significant role in inducing transformation into other cell types. Nuclear reprogramming offers applications in reproductive cloning for livestock propagation and regenerative medicine — developing patient-specific cells for...
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The Central Dogma01:20

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The central dogma explains the flow of genetic information from DNA nucleotides to the amino acid sequence of proteins.
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Crop cultivation has a long history in human civilization, with records showing the cultivation of cereal plants beginning at around 8000 BC. This early plant breeding was developed primarily to provide a steady supply of food.
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Related Experiment Video

Updated: Jul 1, 2025

Non-Viral Engineering of Primary Human T Cells via Homology-Mediated End-Joining Targeted Integration of Large DNA Templates
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[GENE THERAPY: FROM TECHNOLOGY TO REALITY].

Eithan Galun1

  • 1Gene Therapy Institute, Hadassah Medical Center of Jerusalem.

Harefuah
|March 3, 2024
PubMed
Summary
This summary is machine-generated.

Gene therapy has advanced significantly, with approved treatments for various diseases. This review explores CAR-T cells, CRISPR-Cas, and RNAi technologies, addressing challenges in delivery, immunity, and cost.

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Area of Science:

  • Biotechnology
  • Molecular Biology
  • Genetics

Background:

  • Gene therapy has achieved notable success across metabolic, oncologic, infectious, and regenerative medicine.
  • Regulatory agencies in the USA and Europe have approved multiple gene therapy treatments for clinical use.
  • Significant challenges remain, including efficient delivery systems, immunological responses, and high costs.

Approach:

  • This review focuses on three rapidly advancing gene therapy technologies: CAR-T cell therapy, CRISPR-Cas gene editing, and RNA interference (RNAi).
  • The efficacy of genetic vaccination using RNA in synthetic delivery systems, highlighted during the COVID-19 pandemic, informs current advancements.
  • The review examines the potential and challenges associated with these cutting-edge therapeutic modalities.

Key Points:

  • Chimeric Antigen Receptor (CAR)-T cell therapy offers a promising approach for cancer treatment by engineering T cells to target specific antigens.
  • CRISPR-Cas systems provide precise genome editing capabilities, opening new avenues for treating genetic disorders.
  • RNA interference (RNAi) utilizes small RNA molecules to regulate gene expression, offering therapeutic potential for various diseases.

Conclusions:

  • CAR-T cells, CRISPR-Cas, and RNAi represent key frontiers in gene therapy, with the potential to revolutionize medicine.
  • Overcoming challenges in delivery, immunogenicity, and cost is crucial for the widespread clinical adoption of these technologies.
  • Continued research and development in these areas are essential for realizing the full therapeutic promise of gene therapy.