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Artículos vinculados a este trabajo por autores compartidos, revista y gráfico de citas.

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Claudio Bordignon.

Molecular therapy : the journal of the American Society of Gene Therapy·2026
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Tumor-targeted interferon-α gene therapy for glioblastoma: a phase 1 trial.

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Nanoengineered 3D culture substrate enables superior persistence and polyclonal engraftment of genetically engineered hematopoietic stem cells.

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TIGIT disruption rescues the antitumor activity of low avidity TCR-engineered T cells by increasing TCR signal strength.

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Video Experimental Relacionado

Updated: Mar 31, 2026

Defining Gene Functions in Tumorigenesis by Ex vivo Ablation of Floxed Alleles in Malignant Peripheral Nerve Sheath Tumor Cells
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La terapia génica vuelve al centro de la escena

Luigi Naldini1,2

  • 1San Raffaele Telethon Institute for Gene Therapy (TIGET), San Raffaele Scientific Institute, 20132 Milan, Italy.

Nature
|October 16, 2015
PubMed
Resumen
Este resumen es generado por máquina.

Los ensayos clínicos de terapia génica muestran beneficios y seguridad significativos, ofreciendo curas potenciales para afecciones graves. Los avances en el diseño de vectores, la edición genética, la terapia con células madre y la inmunoterapia están impulsando este progreso.

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Área de la Ciencia:

  • Ciencias Biomédicas
  • La genética
  • Medicamentos para la salud

Sus antecedentes:

  • La terapia génica ha prometido durante mucho tiempo curas para enfermedades debilitantes.
  • Los ensayos clínicos recientes demuestran beneficios terapéuticos significativos.
  • En ensayos recientes se ha establecido un excelente historial de seguridad.

Objetivo del estudio:

  • Para resaltar el potencial terapéutico de la terapia génica.
  • Para subrayar la seguridad y eficacia demostradas en ensayos clínicos.
  • Para discutir los avances tecnológicos que impulsan la revitalización de la terapia génica.

Principales métodos:

  • Diseños de vectores mejorados para la entrega de genes dirigidos.
  • Tecnologías de edición de genes para corregir mutaciones.
  • El compromiso de las células madre para la regeneración del tejido.
  • Explotación de las respuestas inmunes para la terapia del cáncer.

Principales resultados:

  • Beneficios terapéuticos notables observados en ensayos clínicos.
  • Excelente registro de seguridad establecido.
  • Pruebas que apoyan la terapia génica como un enfoque curativo para afecciones graves.

Conclusiones:

  • La terapia génica se está dando cuenta de su potencial para curar enfermedades graves.
  • Los avances en los vectores de entrega, la edición de genes, las células madre y la inmunoterapia son clave.
  • El campo de la terapia génica está experimentando una revitalización significativa.