Gene Therapy and Genome Engineering

Gene therapy and genome engineering represent transformative approaches in regenerative medicine that aim to correct, replace, or regulate defective genes responsible for a wide range of inherited and acquired diseases. This session will explore the latest advancements in gene delivery systems, including viral vectors, non-viral carriers, nanoparticles, and advanced genome editing tools such as CRISPR-Cas systems, base editing, and prime editing technologies. Participants will gain insights into how precise genetic modifications are being used to develop novel therapies for genetic disorders, cancer, neurodegenerative diseases, cardiovascular conditions, and metabolic syndromes. The session will also highlight the integration of gene therapy with stem cell technology and tissue engineering to enhance regenerative outcomes and achieve long-term therapeutic benefits. Key discussions will include challenges related to targeting efficiency, off-target effects, immune responses, safety concerns, ethical considerations, and regulatory frameworks governing gene-based interventions. Advances in personalized gene therapy, precision medicine approaches, and in vivo versus ex vivo gene editing strategies will also be addressed. By bringing together researchers, clinicians, bioengineers, and industry experts, this session aims to foster collaboration and innovation, accelerating the development of safe, effective, and next-generation gene and genome engineering therapies that have the potential to revolutionize modern healthcare.

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