Genetic Engineering Advance | Gene Therapy and Clinical Applications by Diljeet Singh | Learn Smarter
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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

  • 3

    Gene Therapy And Clinical Applications

    This section explores gene therapy's principles, its clinical applications, and successful case studies.

    Learning Practice

  • 3.1

    Description

    Learning

  • 3.2

    Learning Objectives

    This section outlines the learning objectives for understanding gene therapy and its clinical applications.

    Learning Practice

  • 3.1

    Section 1: Fundamentals Of Gene Therapy

    Learning

  • 3.1.1

    Gene Therapy

    Gene therapy involves the introduction of genetic material to treat diseases, differentiating between somatic and germline types.

    Learning Practice

  • 3.1.2

    Somatic Gene Therapy

    Somatic gene therapy is a medical technique that modifies the genes in body cells to treat diseases, focusing on individual patient care without heritability.

    Learning Practice

  • 3.1.3

    Germline Gene Therapy

    Germline gene therapy involves altering genes in eggs and sperm, resulting in heritable changes in an individual’s genetic makeup.

    Learning Practice

  • 3.2

    Section 2: Gene Delivery Methods In Therapy

    Learning

  • 3.2.1

    Viral Vectors

    Viral vectors are modified viruses used to deliver therapeutic genes for long-term expression in gene therapy.

    Learning Practice

  • 3.2.2

    Non-Viral Methods

    Non-viral methods of gene delivery provide alternative strategies for introducing genetic material into cells with reduced immune response.

    Learning Practice

  • 3.2.3

    Ex Vivo Gene Therapy

    Ex vivo gene therapy involves modifying cells outside the body before introducing them back into the patient for therapeutic benefits.

    Learning Practice

  • 3.2.4

    In Vivo Gene Therapy

    In Vivo gene therapy involves the direct delivery of genetic material into a patient's body to treat disease.

    Learning Practice

  • 3.3

    Section 3: Clinical Applications Of Gene Therapy

    Learning

  • 3.3.1

    Leber’s Congenital Amaurosis (Lca)

    Leber’s Congenital Amaurosis (LCA) represents a significant genetic condition affecting vision, treated with gene therapy.

    Learning Practice

  • 3.3.2

    Spinal Muscular Atrophy (Sma)

    This section focuses on Spinal Muscular Atrophy and its treatment through the innovative gene therapy Zolgensma.

    Learning Practice

  • 3.3.3

    Hemophilia

    Hemophilia is a genetic disorder where blood does not clot properly, making it essential to explore gene therapy strategies for treatment.

    Learning Practice

  • 3.3.4

    Cancer (Car-T Therapy)

    CAR-T therapy is an innovative gene therapy approach that modifies a patient's T cells to target and destroy cancer cells.

    Learning Practice

  • 3.3.5

    Cystic Fibrosis

    This section discusses cystic fibrosis (CF), its underlying genetic causes, and how gene therapy aims to treat CF by targeting the CFTR gene.

    Learning Practice

  • 3.4

    Section 4: Ethical And Regulatory Aspects

    Learning

  • 3.4.1

    Informed Consent

    Informed consent is a crucial component in gene therapy, requiring patients to understand the risks and objectives involved in their treatment.

    Learning Practice

  • 3.4.2

    Long-Term Safety

    Long-term safety monitoring is crucial for gene therapy to evaluate the potential long-term effects of genetic interventions.

    Learning Practice

  • 3.4.3

    Germline Editing Bans

    This section discusses the ethical implications and regulatory landscape surrounding germline editing bans in various countries.

    Learning Practice

  • 3.4.4

    Regulatory Bodies

    This section overview the essential regulatory bodies involved in gene therapy oversight.

    Learning Practice

  • 3.5

    Section 5: Emerging Trends In Clinical Gene Therapy

    Learning

  • 3.5.1

    Crispr In Clinical Trials

    This section discusses the application of CRISPR technology in current clinical trials aimed at treating genetic disorders like Sickle Cell Disease and Beta Thalassemia.

    Learning Practice

  • 3.5.2

    Personalized Gene Therapies

    Personalized gene therapies utilize genomic information to tailor treatments for individuals, improving the effectiveness of gene therapy.

    Learning Practice

  • 3.5.3

    Ai-Assisted Target Discovery And Delivery Optimization

    This section discusses the role of AI in enhancing the processes involved in gene therapy, particularly focusing on target discovery and optimizing delivery systems.

    Learning Practice

  • 3.5.4

    Gene Silencing Using Sirna And Antisense Oligonucleotides

    This section discusses the mechanisms and applications of gene silencing through siRNA and antisense oligonucleotides.

    Learning Practice

  • 3.6

    Chapter Summary

    This chapter summarizes the principles, challenges, and future directions of gene therapy in treating human diseases.

    Learning Practice

References

Chapter 3_ Gene Therapy and Clinical Applications.pdf

Class Notes

Memorization

What we have learnt

  • Gene therapy aims to correc...
  • Viral and non-viral deliver...
  • Several gene therapies are ...

Final Test

Revision Tests