Identification of the Target Gene: Identifying the specific gene that is mutated and responsible for the disease. Gene Editing or Replacement: Using techniques like CRISPR-Cas9, Zinc Finger Nucleases (ZFNs), or TALENs to edit or replace the defective gene. Delivery to Target Cells: Employing vectors such as viral vectors (e.g., retroviruses, adenoviruses) or non-viral methods (e.g., liposomes, nanoparticles) to deliver the genetic material to the affected cells. Expression and Monitoring: Ensuring that the introduced gene is expressed correctly and monitoring the patient's response to the therapy.
