A revolutionary approach in healthcare aimed at treating or preventing diseases by modifying or replacing faulty genes within an individual’s cells.
A delivery system used in gene therapy to transport therapeutic genes into target cells, often derived from viruses that have been modified to be safe and effective.
The therapeutic gene introduced into the patient’s cells during gene therapy, intended to correct or replace a malfunctioning gene.
The process of precisely modifying an individual’s DNA sequence using techniques such as CRISPR-Cas9, enabling targeted corrections of genetic defects.
A gene therapy approach where cells are removed from the patient’s body, genetically modified in the laboratory, and then reintroduced into the patient.
Gene therapy administered directly into the patient’s body, targeting specific tissues or organs to deliver therapeutic genes.
The body’s defense mechanism against foreign substances, which can pose challenges in gene therapy by targeting and neutralizing viral vectors or modified cells.
A healthcare professional specialized in assessing and communicating genetic information to individuals and families considering gene therapy or genetic testing.
Unintended changes or consequences resulting from gene therapy, such as unintended genetic alterations or immune responses against non-target cells.
A field of medicine that encompasses gene therapy and other approaches aimed at repairing, replacing, or regenerating damaged tissues or organs using biological materials or cells.