RESUMEN

Gene delivery vectors that do not rely on host cell genome integration offer several advantages for gene transfer, chiefly the avoidance of insertional mutagenesis and position effect variegation. However, unless engineered for replication and segregation, nonintegrating vectors will dilute progressively in proliferating cells, and are not exempt of epigenetic effects. This article provides an overview of the main nonintegrating viral (adenoviral, adeno-associated viral, integration-deficient retro-lentiviral, poxviral), and nonviral (plasmid vectors, artificial chromosomes) vectors used for preclinical and clinical cell and gene therapy applications. Particular emphasis is placed on their use in hematologic disease.

Asunto(s)

Terapia Genética , Vectores Genéticos/genética , Adenoviridae/genética , Animales , Ensayos Clínicos como Asunto/historia , Dependovirus/genética , Edición Génica , Expresión Génica , Técnicas de Transferencia de Gen , Terapia Genética/efectos adversos , Terapia Genética/historia , Terapia Genética/métodos , Vectores Genéticos/clasificación , Historia del Siglo XX , Historia del Siglo XXI , Humanos , Plásmidos/genética , Poxviridae/genética , Transducción Genética