Gene therapy breakthrough

• Autores: Andy Coghlan
• Localización: New scientist, ISSN 0262-4079, Nº. 3115, 2017, pág. 8
• Idioma: inglés
• Texto completo no disponible (Saber más ...)
• Resumen

  • A teenage boy with an inherited disease that affects millions worldwide seems to have been cured using gene therapy. The treatment appears to have stopped the painful symptoms of sickle cell disease, demonstrating the potential for gene therapy to treat common genetic diseases. The idea of gene therapy--using strands of DNA to compensate for a person's malfunctioning genes--is almost three decades old. However, the approach has so far mostly been used to treat very rare diseases. Here, Coghlan discusses how gene therapy treats common genetic disorder