Gene therapy

Gene Therapy targets the genetic root causes of diseases by directly modifying individuals' DNAs. This field leverages cutting-edge techniques to correct or replace defective genes responsible for various genetic disorders and conditions.

Applications include:

• Gene delivery systems: the delivery of therapeutic genes into the patient’s cells via viral vectors
• Gene editing technologies: advanced gene editing tools like CRISPR-Cas9, TALENs (Transcription Activator-Like Effector Nucleases), and ZFNs (Zinc Finger Nucleases) are employed to make precise changes to the genome.
• In Vivo and Ex Vivo approaches: gene therapy can be administered through in vivo methods, where genes are delivered directly into the patient’s body, or ex vivo methods, where cells are extracted, genetically modified outside the body, and then reintroduced into the patient
• Cell and tissue specificity: development of targeted gene delivery methods to ensure that therapeutic genes are expressed in the specific cells or tissues affected by the disease.