Functional Genomics and Gene Therapy for Rare Diseases

Functional genomics aims to understand the roles of genes and their interactions, providing critical insights for rare disease treatment. Gene therapy offers the potential to correct genetic abnormalities, with viral vector-based delivery and CRISPR technology paving the way for innovative solutions. Clinical trials focus on ensuring the safety, efficacy, and durability of these treatments. As research progresses, refining delivery methods and addressing ethical considerations remain essential for wider adoption.

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