Despite its potential, gene therapy faces several challenges:
Delivery Efficiency: Ensuring that the genetic material is delivered to the correct cells and tissues in sufficient quantities. Immune Response: Avoiding immune system reactions to the viral vectors or introduced genes. Off-Target Effects: Minimizing unintended alterations to other parts of the genome, which could cause harmful side effects. Long-term Expression: Ensuring that the introduced gene remains functional and expresses the desired protein over the long term. Ethical Concerns: Addressing ethical issues related to genetic modifications, particularly germline alterations that can be passed down to future generations.
