genbehandling

Genbehandling, or gene therapy, is a medical approach that aims to treat or prevent disease by introducing, removing, or altering genetic material within a patient's cells. The goal can be to correct a faulty gene, compensate for a loss of function, or enhance cellular performance to combat disease. Gene therapy can involve altering DNA in living cells or modulating gene expression without changing the underlying DNA sequence.

Therapies are generally categorized as ex vivo and in vivo. In ex vivo approaches, cells are removed, genetically modified outside the body, and then returned to the patient. In vivo approaches deliver genetic material directly to the patient’s tissues. Techniques include gene replacement or addition, gene silencing, and genome editing to correct or disable disease-causing mutations.

Delivery methods rely on vectors and editing tools. Viral vectors such as adeno-associated virus, lentivirus, and adenovirus are commonly used to transport genetic material into cells, while non-viral methods include lipid nanoparticles and physical delivery methods. Genome editing tools, notably CRISPR-Cas9 and related systems, enable precise modifications to the genome, whereas non-editing approaches add functional gene copies to restore normal activity.

Applications span inherited monogenic disorders, certain cancers through engineered immune cells, and other diseases where correcting gene function can alter disease progression. Several gene therapies have received regulatory approvals in different regions, reflecting progress alongside ongoing research and development. Safety considerations include potential off-target edits, immune responses, and insertional mutagenesis, as well as long-term effects and manufacturing costs. Ethical and access issues also shape policy and clinical use.