abstract

Despite these advancements, gene therapy faces several challenges. Immune responses to viral vectors, the potential for insertional mutagenesis, and difficulties in delivering the therapy to specific tissues are major hurdles that need to be addressed. Moreover, the high cost of gene therapy and ethical concerns surrounding genetic modifications continue to spark debate. Clinical outcomes have been promising, with successful treatments reported for conditions conditions like haemophilia, some cancers, and spinal muscular atrophy are examples. Nevertheless, in order to have a complete understanding of the effectiveness and safety of these therapies. As research continues, overcoming the existing challenges vital to the expansion of gene therapy's potential uses in clinical settings.