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Advancements in Gene Therapy: A Rapidly Evolving Frontier

NextEdge Admin

05 Mar 2024

3 min 25 sec

Gene therapy, a rapidly advancing field, seeks to enhance genes by rectifying mutations or making precise modifications for therapeutic purposes. Its primary goal is to improve genetic function by correcting mutations or performing site-specific modifications to address various medical conditions.

The challenge lies in delivering therapeutic genes into stem cells, a critical process where molecular carriers, known as vectors, play a crucial role. These vectors must exhibit specificity, efficiency, immune system evasion, environmental safety, and the ability to sustain long-term gene expression.

ROCTAVIAN utilizing adeno-associated virus serotype 5 as vectors represents a singular gene therapy designed for the treatment of severe hemophilia A in adults lacking antibodies to the AAV5 virus, as confirmed by a blood test. Employing a modified virus as a vector, ROCTAVIAN facilitates the delivery of a functional Factor VIII gene to liver cells. This process enables the body to independently produce clotting factor, mitigating the risk of bleeding occurrences. It is crucial to note that the modified virus utilized in ROCTAVIAN is devoid of viral DNA, ensuring it does not induce diseases in humans. This innovative therapy offers a one-time intervention to empower the body’s natural ability to clot blood effectively, providing a transformative approach to managing hemophilia A.

The field took an exciting turn with CRISPR, a revolutionary tool facilitating precise editing of DNA sequences. Derived from a bacterial immune system, CRISPR technology involves Cas9 (a nuclease), RNA guide, and target DNA, allowing targeted genome editing. A remarkable application of CRISPR is seen in the treatment of genetic disorders like sickle cell anemia, where it can precisely modify the genetic code within hematopoietic stem cells to address the underlying mutation. The Food and Drug Administration (FDA) has granted approval for Casgevy recently, a groundbreaking gene-editing therapy designed to treat sickle cell disease.

In essence, gene therapy holds the potential to revolutionize healthcare by addressing genetic and acquired diseases at their roots. As technology advances and ethical considerations are addressed, the future may witness routine genetic interventions, contributing to the pursuit of healthier and more resilient human populations.