Question 1

What is the Meaning of Gene Therapy?

Accepted Answer

Gene therapy is a medical intervention that modifies a person's genes to treat or prevent disease. This therapeutic strategy addresses the root cause of genetic disorders by altering cellular genetic material, moving beyond symptomatic management to biological correction.

Question 2

What is the Context of Gene Therapy?

Accepted Answer

This intervention operates at cellular and molecular levels within the human body. Gene therapy targets specific cells or tissues where a genetic defect is present or a therapeutic protein is needed, influencing various physiological systems. It directly impacts cellular machinery responsible for protein synthesis.

Question 3

What is the Significance of Gene Therapy?

Accepted Answer

Its clinical importance lies in its potential to offer durable or curative treatments for conditions previously considered untreatable. This approach can transform patient outcomes for inherited disorders, certain cancers, and chronic diseases, shifting focus from management to correction. It provides a new avenue for addressing fundamental biological dysfunctions.

Question 4

What is the Mechanism of Gene Therapy?

Accepted Answer

The mechanism involves delivering genetic material, typically DNA or RNA, into target cells using a vector, often a modified virus. Once inside, the introduced material can correct a faulty gene, replace a missing gene, or introduce a new gene producing a therapeutic protein. This restores normal cellular function, altering cellular protein production.

Question 5

What is the Application of Gene Therapy?

Accepted Answer

Gene therapy is currently applied in clinical practice for severe genetic conditions, including spinal muscular atrophy, inherited retinal dystrophies, and severe combined immunodeficiency. Its utility extends to some hematological disorders and is under investigation for broader applications in metabolic and neurodegenerative conditions. The approach requires careful patient selection.

Question 6

What is the Metric of Gene Therapy?

Accepted Answer

Effectiveness and safety are monitored through clinical and laboratory assessments. Measurements include evaluating persistence of delivered genetic material, assessing expression levels of the therapeutic gene product, and tracking changes in disease-specific biomarkers. Clinical improvements and functional assessments also serve as crucial indicators. Immune responses are closely observed.

Question 7

What is the Risk of Gene Therapy?

Accepted Answer

Potential risks include immune reactions against the viral vector or therapeutic gene product, which can reduce efficacy or cause adverse events. Off-target gene insertion might disrupt other genes or lead to unintended cellular changes, though vector design improvements mitigate this. Long-term durability and potential for late-onset complications remain areas of ongoing study.

Gene Therapy

Meaning

Context

Significance

Mechanism

Application

Metric

Risk

Can International Standards Harmonize Regulatory Approaches for Advanced Therapies?