Gene Therapy

Introduction

Gene therapy replaces a faulty gene or adds a new gene in an attempt to cure a disease or improve your body’s ability to fight a disease.

Definition

Gene therapy is a technique that modifies a person’s genes to treat or cure a disease. Gene therapies can work by several mechanisms such as:

  • Replacing a disease-causing gene with a healthy copy of the gene.
  • Inactivating a disease-causing gene that is not functioning properly.
  • Introducing a new or modified gene into the body to help treat a disease.
Indications
  • Cancer.
  • Genetic diseases.
  • Infectious diseases.
Types
TypeFormation  
Plasmid DNACircular DNA molecules can be genetically engineered to carry therapeutic genes into human cells.  
Viral vectorsViruses have a natural ability to deliver genetic material into cells, and therefore some gene therapy products are derived from viruses.  
Bacterial vectorsBacteria can be modified to prevent them from causing infectious disease.  
Human gene editing technology  To disrupt harmful genes or to repair mutated genes.
Patient-derived cellular gene therapy products  Cells are removed from the patient, genetically modified (often using a viral vector), and then returned to the patient.

There are mainly two approaches for the transfer of genes in gene therapy:

  1. Transfer of genes into the patient’s cells outside the body (ex vivo gene therapy).
  2. Transfer of genes directly to cells inside the body (in vivo).
Ex Vivo Gene Therapy

In this mode of gene therapy, genes are transferred to the cells grown in culture, and transformed cells are selected, multiplied, and then introduced into the patient.

In Vivo Gene Therapy

The in vivo method of gene transfer involves the transfer of cloned genes directly into the tissues of the patient.

Advantages
  • By preventing the transmission of disease genes, the expense and risk of somatic cell therapy for multiple generations is avoided.
  • Medicine should respond to the reproductive health needs of prospective parents at risk for transmitting serious genetic diseases.
Disadvantages
  • Gene therapy is very expensive and will never be cost-effective enough to merit high social priority.
  • Germline gene therapy would violate the rights of subsequent generations to inherit a genetic endowment that has not been intentionally modified.
Roles of a Nurse in Gene Therapy
  • Provides anticipatory guidance.
  • Assures informed decision making/consent.
  • Assures privacy and confidentiality of genetic information.
  • Develops treatment and management plans.
  • Administers gene therapy.
  • Observes patients for expected and unexpected side effects of treatment, including psychosocial and emotional responses.
  • Participates in developing long-term follow-up plans.
  • Serves as an information source to patients, families, and the public.
  • Provides relevant, accurate, and understandable information to patients, in both written and verbal forms.
  • Assures that all patient/family questions are answered Advocate
Documentation
  • Document the date, time, mode of therapy provided to the patient, duration of procedures, and condition of the patient before and after the procedure.
  • Document if any side effects were experienced.
Special Consideration
  • Continuous monitoring is required to assess potential unintended genetic changes.
  • Gene therapy raises concerns about genetic modifications, accessibility, and affordability.
  • Gene therapy products undergo rigorous approval processes to ensure safety.
  • Not all patients are eligible; factors like genetic compatibility and disease severity are considered.
  • Patients require extended monitoring to track efficacy and adverse effects.
  • Viral Vectors vs. Non-Viral Methods – Choosing the right delivery system affects efficacy and safety.
  • Technologies like CRISPR allow precise modifications but require careful validation.
  • In Pediatric Patients – Early intervention may be beneficial, but long-term effects must be studied.
  • In Geriatric Patients – Age-related factors may influence gene therapy effectiveness.
  • In Rare Genetic Disorders – Gene therapy offers hope for previously untreatable conditions.

REFERENCES

  1. Annamma Jacob, Rekha, Jhadav Sonali Tarachand: Clinical Nursing Procedures: The Art of Nursing Practice, 5th Edition, March 2023, Jaypee Publishers, ISBN-13: 978-9356961845 ISBN-10: 9356961840
  2. Omayalachi CON, Manual of Nursing Procedures and Practice, Vol 1, 3 Edition 2023, Published by Wolters Kluwer’s, ISBN: 978-9393553294
  3. Sandra Nettina, Lippincott Manual of Nursing Practice, 11th Edition, January 2019, Published by Wolters Kluwers, ISBN-13:978-9388313285
  4. Adrianne Dill Linton, Medical-Surgical Nursing, 8th Edition, 2023, Elsevier Publications, ISBN: 978-0323826716
  5. Donna Ignatavicius, Medical-Surgical Nursing: Concepts for Clinical Judgment and Collaborative Care, 11th Edition ,2024, Elsevier Publications, ISBN: 978-0323878265
  6. Lewis’s Medical-Surgical Nursing, 12th Edition,2024, Elsevier Publications, ISBN: 978-0323789615
  7. AACN Essentials of Critical Care Nursing, 5th Ed. Sarah. Delgado, 2023, Published by American Association of Critical-Care Nurses ISBN: 978-1264269884
  8. Ernstmeyer K, Christman E, editors. Nursing Fundamentals [Internet]. 2nd edition. Eau Claire (WI): Chippewa Valley Technical College; 2024. PART IV, NURSING PROCESS. Available from: https://www.ncbi.nlm.nih.gov/books/NBK610818/

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