Gene therapy: Myth or reality?
C R Biol
; 339(7-8): 314-8, 2016.
Article
em En
| MEDLINE
| ID: mdl-27260498
ABSTRACT
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.
Palavras-chave
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
Texto completo:
1
Base de dados:
MEDLINE
Assunto principal:
Terapia Genética
Idioma:
En
Ano de publicação:
2016
Tipo de documento:
Article