What Are the Challenges of Gene Therapy in Neonates?
While gene therapy holds great promise, it also faces several challenges:
- Delivery: Ensuring that the therapeutic genes reach the affected cells and tissues in neonates can be complex. - Safety: There are concerns about the potential for immune reactions or insertional mutagenesis, which could cause unintended genetic changes. - Durability: The long-term effectiveness of gene therapy remains uncertain, and there may be a need for repeat treatments.
