[Gene therapy: current situation and expectations]. / Terapia génica: realidades actuales y expectativas.

In this review the current situation of gene therapy is described in hematological diseases, immunological conditions, and cancer. In all of them, the principal objective of various approaches with gene therapy is transduction of therapeutic genes in most of target cells. In chronic or immunological diseases, a stable expression of therapeutic genes is also required; in tumor cells, the efficiency or percentage of transduced cells make conditional on the treatment success. Consequently, vectors are one of the basic elements to optimize gene therapy approaches and protocols in view of the facts that we know that with liposomes less than 10% of cells are transduced, that retrovirus only infect cells in replication, and that adenovirus give rise to an important inflammatory response and a transitory transduction of the therapeutic gene. In addition recent approaches in cancer gene therapy with selective replication virus, suicidal genes, etc., are discussed.

Terapia génica: realidades actuales y expectativas / Gene therapy: current situation and expectations

En esta revisión se describe la situación actual de la terapia génica en enfermedades hematológicas, inmunológicas y en cáncer. En todas ellas el objetivo principal de los diversos abordajes de la terapia génica es el transducir los genes terapéuticos en la mayor parte de las células diana. A nivel de enfermedades crónicas o inmunológicas se requiere, asimismo, una expresión estable de los genes terapéuticos y a nivel de las células tumorales la eficiencia o porcentaje de células transducidas condiciona el éxito de los tratamientos. Por consiguiente, los vectores son uno de los elementos básicos para optimizar los abordajes y protocolos de terapia génica dado que sabemos que con el empleo de liposomas menos del 10% de las células van a ser transducidas, que con el empleo de retrovirus sólo se van a infectar células en replicación y que con los adenovirus va a haber una respuesta inflamatoria importante y una transducción transitoria del gen terapéutico. Asimismo se discuten los últimos abordajes en terapia génica del cáncer con virus de replicación selectiva, genes suicidas, etc

In this review the current situation of gene therapy is described in hematological diseases, immunological conditions, and cancer. In all of them, the principal objective of various approaches with gene therapy is transduction of therapeutic genes in most of target cells. In chronic or immunological diseases, a stable expression of therapeutic genes is also required; in tumor cells, the efficiency or porcentage of transduced cells make conditional on the treatment success. Consequently, vectors are one of the basic elements to optimize gene therapy approaches and protocols in view of the facts that we know that with liposomes less than 10% of cells are transduced, that retrovirus only infect cells in replication, and that adenovirus give rise to an important inflammatory response and a transitory transduction of the therapeutic gene. In addition recent approaches in cancer gene therapy with selective replication virus, suicidal genes, etc., are discussed