Gene therapy: Myth or reality?
Gene therapy has become a reality, although still a fragile one. Clinical benefit has been achieved over the last 17years in a limited number of medical conditions for which pathophysiological studies determined that they were favorable settings. They include inherited disorders of the immune system, leukodystrophies, possibly hemoglobinopathies, hemophilia B, and retinal dystrophies. Advances in the treatment of B-cell leukemias and lymphomas have also been achieved. Advances in vector development and possible usage of gene editing may lead to significant advances over the next years.
Paris Descartes-Sorbonne Paris Cité University, Imagine Institute, 75015 Paris, France; Immunology and Pediatric Hematology Department, Assistance publique-Hôpitaux de Paris, 75015 Paris, France; Inserm UMR 1163, 75015 Paris, France; Collège de France, 75005 Paris, France. Electronic address: alain.fischer@inserm.fr.
Alain
A
Fischer
D000818:Animals; D000229:Dependovirus / Q000235:genetics; D018014:Gene Transfer Techniques; D030342:Genetic Diseases, Inborn / Q000235:genetics / Q000628:therapy; D015316:Genetic Therapy; D005822:Genetic Vectors; D006801:Humans; D009369:Neoplasms / Q000628:therapy
Adeno-associated viruses; B-cell leukemia; Chimeric antigen receptors (CAR); Gene editing; Gene therapy; Immunodéficience combinée sévère; Leucémie des cellules B; Retroviruses; Récepteur d’antigène chimérique; Rétrovirus; Severe combined immunodeficiency; Thérapie génique; Virus adéno-déficient; Édition de gène