Journal of Genetics and Gene Therapy

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Short Communication, J Genetic Gene Ther Vol: 7 Issue: 3

Gene Therapy: Transforming the Future of Medicine through Genetic Intervention

Adler Hong*

1Department of Medicine, University of California, San Diego, USA

*Corresponding Author: Adler Hong,
Department of Medicine, University of California, San Diego, USA
E-mail: hong.a@gmail.com

Received date: 02 September, 2023, Manuscript No. JGGT-23-117820;

Editor assigned date: 04 September, 2023, PreQC No. JGGT-23-117820 (PQ);

Reviewed date: 18 September, 2023, QC No. JGGT-23-117820;

Revised date: 25 September, 2023, Manuscript No. JGGT-23-117820 (R);

Published date: 05 October, 2023, DOI: 10.4172/Jggt.1000155.

Citation: Hong A (2023) Gene Therapy: Transforming the Future of Medicine through Genetic Intervention. J Genetic Gene Ther 7:3.

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Description

Gene therapy represents a significant area that is fundamental within the area of medical science. It provides treatment and potentially preventing a wide range of genetic disorders by harnessing the power of genetics to modify or replace malfunctioning genes. Gene therapy is a medical technique that involves the alteration, addition, or replacement of genes within an individual's cells to treat or prevent disease. It addresses the fundamental causes of genetic disorders by focusing on the underlying genetic mutations or abnormalities responsible for the condition [1,2].

Historical development of gene therapy

The field of gene therapy has evolved significantly over time, with significant milestones:

Early concepts: The information of gene therapy was first conceptualized in the 1960s, and early experiments in animals followed instantaneously following that occur.

First human trials: The first clinical trials in humans began in the 1980s. These initial efforts focused on addressing Severe Combined Immunodeficiency (SCID), a common genetic disease.

Challenges and setbacks: The field faced significant setbacks in the late 1990s, including the death of a patient in a clinical trial and technical challenges in gene delivery methods.

Emergence of viral vectors: The development of less hazardous and more effective vectors based on viruses for gene delivery was an important time in the field and generating a significant increase in clinical studies and research.

FDA approvals: In recent years, the U.S. Food and Drug Administration (FDA) have granted approvals for several gene therapy products to treat genetic disorders, including spinal muscular atrophy and certain forms of inherited blindness [3-5].

Principles of gene therapy

Gene therapy operates on several core principles that support its technique to modifying genetic material:

Genetic modification: Gene therapy focuses on the modification of the Genetic Material (DNA) within an individual's cells. This can involve adding, repairing, or replacing genes.

Delivery systems: Various delivery systems, such as viral vectors or non-viral methods, are employed to transport therapeutic genes into target cells.

Specific targeting: Precision is essential in gene therapy to ensure that therapeutic genes are delivered to the right cells and tissues without affecting healthy ones.

Safety and efficacy: Ensuring the safety and efficacy of gene therapy is paramount. Extensive preclinical testing and clinical trials are conducted to assess the therapy's benefits and potential risks.

Ethical considerations: Ethical considerations in gene therapy encompass issues such as informed consent, privacy, and the responsible use of genetic modification.

Therapeutic techniques in gene therapy

Gene therapy encompasses several therapeutic methods aimed at addressing different types of genetic disorders:

Gene replacement therapy: In some conditions where a particular gene is missing or non-functional, gene replacement therapy aims to introduce a functional copy of the gene to restore regular cellular function [6,7].

Gene editing: Gene editing techniques, such as CRISPR-Cas9, enable precise modifications to the genetic code by adding, deleting, or altering specific sequences within the DNA.

Gene augmentation: In some cases where genes are present but not producing enough functional protein, gene augmentation therapy requires to increase the production of the deficient protein.

Cell-based gene therapy: In some instances, gene therapy involves the modification of a patient's own cells outside the body before reintroducing them to treat conditions such as certain cancers or inherited blood disorders [8].

Applications of gene therapy

Gene therapy has made significant advances in treating various genetic disorders and has broad applications:

Hematological disorders: Gene therapy has shown excellent possibilities in treating inherited blood disorders such as betathalassemia and sickle cell anemia. For example, the FDA approved the first gene therapy product for beta-thalassemia in 2019.

Inherited blindness: Inherited forms of blindness, such as Leber congenital amaurosis, can be treated with gene therapy. Luxturna, an FDA-approved gene therapy, has restored vision in some patients [9,10].

References

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