Question 1

What is the Meaning of Somatic Gene Therapy?

Accepted Answer

Somatic Gene Therapy is a highly specialized therapeutic technique that involves the introduction of functional genetic material into the non-reproductive cells, known as somatic cells, of a patient for the explicit purpose of treating or preventing a specific disease. Crucially, the genetic change is strictly confined to the treated individual and is not capable of being inherited by their offspring. This method is currently being actively developed to correct congenital hormonal deficiencies or genetic disorders by restoring proper protein function in specific, targeted tissues like muscle, liver, or endocrine glands.

Question 2

What is the Origin of Somatic Gene Therapy?

Accepted Answer

The concept of gene therapy originated in the late 20th century, primarily driven by the clinical desire to treat single-gene disorders by replacing a defective gene with a functional, healthy copy. The term "somatic" was introduced to clearly distinguish it from germline therapy, underscoring the non-heritable nature of the therapeutic intervention. Initial human clinical trials commenced in the 1990s, and the field has achieved significant regulatory milestones in recent years.

Question 3

What is the Mechanism of Somatic Gene Therapy?

Accepted Answer

The typical mechanism involves carefully packaging the therapeutic gene into a specialized delivery vehicle, most often a modified, non-pathogenic virus vector, which is then precisely administered to the patient. The vector is engineered to target the appropriate somatic cells, successfully enters them, and delivers the functional gene into the nucleus. The host cell then utilizes its machinery to express the new, functional protein, thereby correcting the underlying cellular or hormonal defect without affecting the patient's germline.

Somatic Gene Therapy

Meaning

Origin

Mechanism

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