IJMPD | GENE Therapy: A Review

Indexing and Abstracting

GENE Therapy: A Review

Simranjit Kaur , Rajesh Kumar , Ajeet Pal Singh , Amar Pal Singh , Meenakshi Malhotra

International Journal of Medical, Pharmacy and Drug Research(IJMPD), Vol-8,Issue-1, January - March 2024, Pages 26-33 ,

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Article Info: Received: 08 Feb 2024; Received in revised form:15 Mar 2024; Accepted: 22 Mar 2024; Available online: 31 Mar 2024

Abstract:

Gene therapy, a revolutionary approach to treating genetic disorders, holds tremendous promise in offering targeted and potentially curative treatments for a wide range of diseases. This paper provides an overview of the principles, techniques, and recent advancements in gene therapy. It begins with an introduction to the concept of gene therapy, elucidating its underlying mechanisms and various delivery methods, including viral vectors, non-viral vectors, and genome editing tools such as CRISPR-Cas9. The paper then discusses the applications of gene therapy in treating genetic disorders, including but not limited to hemophilia, cystic fibrosis, muscular dystrophy, and certain types of cancer. By correcting or replacing faulty genes, gene therapy has the potential to alleviate symptoms, halt disease progression, and even restore normal cellular function. This therapeutic strategy involves delivering functional genes into target cells to restore normal cellular function. Utilizing viral vectors or non-viral methods, gene therapy can potentially treat a wide range of diseases, including inherited disorders like cystic fibrosis, muscular dystrophy, and hemophilia, as well as certain types of cancer and cardiovascular diseases. Clinical trials have demonstrated both the safety and efficacy of gene therapy in various contexts, although challenges such as immune responses, delivery efficiency, and long-term gene expression persist. Continued research and technological advancements are essential to overcome these hurdles and unlock the full potential of gene therapy as a transformative medical intervention. Furthermore, this paper highlights recent breakthroughs and clinical successes in the field of gene therapy, showcasing notable case studies and trials that demonstrate its efficacy and safety. These advancements underscore the growing momentum and optimism surrounding gene therapy as a viable treatment option for previously incurable conditions. In conclusion, gene therapy represents a paradigm shift in medicine, offering hope for individuals affected by genetic disorders and opening new avenues for personalized and precision medicine. With continued research, collaboration, and investment, gene therapy holds the potential to transform the landscape of healthcare, ultimately improving patient outcomes and quality of life.

Keywords:

Gene Therapy, Genetic Disorders Treatment, Viral Vectors, CRISPR-Cas9, Clinical Trials

References:

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Indexing and Abstracting

GENE Therapy: A Review

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