Gene therapy definition
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Gene Therapy Definition and Core Concepts
Gene therapy is a medical technique that involves introducing, altering, or replacing genetic material within a person's cells to treat or prevent disease. The main goal is to modify gene expression or correct abnormal genes to achieve a therapeutic effect Sayed2022Saadi2021Tang2020+5 MORE. This can be done by adding a healthy copy of a gene, silencing a faulty gene, or editing the genome to fix mutations Sayed2022Tang2020Belete2021+2 MORE.
Types and Methods of Gene Therapy
Gene therapy can be categorized in several ways:
- Gene Addition/Replacement: Supplying a functional gene to compensate for a defective or missing one Sayed2022Tang2020Belete2021+2 MORE.
- Gene Silencing: Using molecules like siRNA or shRNA to reduce or block the expression of harmful genes .
- Gene Editing: Directly correcting mutations in the DNA using tools such as CRISPR/Cas9, zinc-finger nucleases, or TALENs Sayed2022Belete2021Papanikolaou2021+1 MORE.
Gene therapy can be performed:
- In vivo: Directly delivering genetic material into the patient’s body Saadi2021Tang2020.
- Ex vivo: Modifying cells outside the body and then reintroducing them into the patient Saadi2021Tang2020.
Delivery Systems and Vectors in Gene Therapy
The success of gene therapy depends on effective delivery of genetic material into target cells. This is achieved using:
- Viral Vectors: Modified viruses (like adeno-associated virus, adenovirus, and lentivirus) are commonly used because of their high efficiency in delivering genes Sayed2022Saadi2021Li2023+2 MORE.
- Non-Viral Methods: These include physical (electroporation, microinjection), chemical (liposomes, nanoparticles), and biological approaches Sayed2022Saadi2021.
Each method has its own advantages and challenges, such as immune responses or the risk of unintended genetic changes Sayed2022Saadi2021Li2023+2 MORE.
Regulatory and Clinical Perspectives
A modern definition of gene therapy emphasizes the intentional and expected permanent alteration of a specific DNA sequence in the genome for clinical purposes . Regulatory agencies and scientists categorize gene therapy into direct, compensatory, and augmenting types to provide clarity for research and clinical applications .
Gene therapy has been approved for several diseases, including genetic disorders, cancers, and some acquired conditions, with ongoing research to improve safety and effectiveness Tang2020Belete2021Lejman2023+3 MORE.
Conclusion
Gene therapy is a transformative approach that involves the introduction, modification, or replacement of genetic material in cells to treat or prevent disease. It uses a variety of strategies and delivery systems, with ongoing advancements aimed at improving its safety, effectiveness, and range of applications Sayed2022Sherkow2018Saadi2021+7 MORE.
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