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Revolutionizing Medicine: Advances in Gene Therapy for Genetic Disorders


Dr. P. Ravi Sekhar, Dr. Y. Savithri
Pages: 67-76
ISBN: 978-93-5834-440-0


Advances in Biological Sciences and Biotechnology (Volume -7)

Advances in Biological Sciences and Biotechnology
(Volume - 7)

Abstract

Gene therapy represents a revolutionary approach in modern medicine, offering potential cures for a variety of genetic disorders that were once deemed untreatable. This field has made significant strides in recent years, transitioning from experimental stages to clinical applications with profound impacts on patient outcomes. Gene therapy involves the introduction, correction, or removal of genetic material within a patient's cells to rectify genetic abnormalities. Key methods include gene addition, gene editing, RNA interference, and gene knockout or replacement, each tailored to address specific genetic defects. Recent advances, particularly in gene editing technologies like CRISPR-Cas9, have enabled unprecedented precision in correcting genetic mutations. These breakthroughs have paved the way for treating conditions such as severe combined immunodeficiency (SCID), hemophilia, cystic fibrosis, Duchenne muscular dystrophy, Leber congenital amaurosis, and sickle cell disease. Both ex vivo and in vivo delivery methods are being refined to enhance efficiency and safety, utilizing viral and non-viral vectors for targeted gene delivery. Despite the remarkable progress, challenges remain, including ensuring efficient delivery to target cells, minimizing off-target effects, achieving long-term efficacy, and addressing ethical and regulatory considerations. Nonetheless, the ongoing advancements in gene therapy hold promise for transforming the treatment landscape of genetic disorders, moving towards definitive cures and significantly improving patient quality of life. This abstract highlights the key developments and future directions in gene therapy, underscoring its potential to revolutionize medicine and provide hope for patients with genetic disorders.

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© Integrated Publications.
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