Gene Therapy and Clinical Applications
Gene therapy is utilized to rectify or substitute faulty genes in patients, employing innovative methods such as viral and non-viral delivery systems to ensure successful treatment. The chapter emphasizes the approval of several gene therapies for clinical use and addresses the essential ethical, safety, and regulatory issues that accompany this rapidly advancing field. With a focus on personalized approaches and the integration of AI in gene therapy, the future possibilities are promising.
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Sections
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3.1Description
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3.1Section 1: Fundamentals Of Gene Therapy
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3.2Section 2: Gene Delivery Methods In Therapy
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3.3Section 3: Clinical Applications Of Gene Therapy
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3.4Section 4: Ethical And Regulatory Aspects
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3.5Section 5: Emerging Trends In Clinical Gene Therapy
What we have learnt
- Gene therapy aims to correct or replace defective genes in patients.
- Viral and non-viral delivery systems are critical to therapy success.
- Several gene therapies are now approved and in clinical use.
- Ethical issues and safety are central to regulation and public acceptance.
- Future applications include personalized and AI-optimized gene therapies.
Key Concepts
- -- Gene Therapy
- The introduction of genetic material into a patient to treat or prevent disease.
- -- Somatic Gene Therapy
- A type of gene therapy that affects only the treated individual and is widely accepted.
- -- Germline Gene Therapy
- A controversial form of gene therapy that alters genes in reproductive cells, thereby affecting future generations.
- -- Viral Vectors
- Modified viruses used to deliver genes into cells for therapeutic purposes, associated with long-term gene expression.
- -- Ex Vivo Gene Therapy
- Cells are modified outside the body and then reintroduced, often used in CAR-T therapies for cancer.
- -- In Vivo Gene Therapy
- Direct delivery of therapeutic genes to patients' bodies, commonly employed for muscular dystrophy and eye disorders.
- -- Regulatory Bodies
- Organizations responsible for the oversight of gene therapy practices, including the FDA, EMA, and ICMR.
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