Human gene therapy

Human gene therapy is a medical procedure that involves the introduction, removal, or alteration of genetic material within a patient's cells to treat or prevent disease. The therapy is considered a form of genetic engineering and is a rapidly evolving field in biomedicine.

History

The concept of human gene therapy was first proposed during the 1960s. The first successful demonstration of gene therapy in mammals was achieved by Martin Cline in 1980. However, the first therapeutic use of gene transfer in humans did not occur until 1990, under the guidance of William French Anderson.

Types of Gene Therapy

There are two types of gene therapy: somatic gene therapy and germline gene therapy. Somatic gene therapy involves transferring a section of DNA to any cell of the body that doesn't produce sperm or eggs. Effects of this therapy are not inherited. On the other hand, in germline gene therapy, the genes in eggs or sperm are changed, or the changes are made in the genes of an early stage embryo. Effects of this therapy are inheritable.

Techniques

Several techniques are available for human gene therapy, including viral vectors, non-viral methods, and the newer genome editing technologies such as CRISPR-Cas9. Viral vectors are tools commonly used by molecular biologists to deliver genetic material into cells. Non-viral methods include the direct introduction of therapeutic DNA into target cells. CRISPR-Cas9 is a revolutionary gene-editing system that allows geneticists to cut and paste DNA where needed.

Applications

Human gene therapy has been proposed for various diseases, including cancer, genetic disorders, and infectious diseases. It has also been suggested as a possible method for treating aging or age-related disorders.

Ethical and Safety Considerations

Human gene therapy has raised significant ethical and safety concerns. These include issues related to germline therapy, which could potentially change the genes in eggs or sperm and affect future generations. There are also concerns about the potential for unforeseen side effects, including the possibility of causing cancer.

Future

The future of human gene therapy is promising, with ongoing research and clinical trials. However, many challenges remain, including the need for more efficient and safer delivery methods, the high cost of gene therapy, and the ethical and safety concerns mentioned above.

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