Gene Therapy: The Promise of a Permanent Cure

Abstract

Gene therapy offers the possibility of a permanent cure for any of the more than 10,000 human diseases caused by a defect in a single gene. Among these diseases, the hemophilias represent an ideal target, and studies in both animals and humans have provided evidence that a permanent cure for hemophilia is within reach.

Personalized Health Care in 2013: A Status Report on the Impact of Genomics

“…Gene therapy, made possible through recombinant DNA technologies developed in the 1980s, has long offered hope for curing many diseases. Despite the unexpected difficulty of developing practical gene therapies, progress is being made-as Porada and colleagues discuss in their commentary [32]-and gene therapy is seen as a hope for curing many of the perhaps 10,000 human diseases that are caused by defects in a single gene.…”

Induced Pluripotent Stem Cells (iPSCs) and Gene Therapy: A New Era for the Treatment of Neurological Diseases

“…Sadly, no efficient pharmacological treatment is known. To this aim, gene therapy has the potential to represent an effective and permanent cure [11,12]. However, replacing damaged cells with engineered patient-specific iPSCs able to integrate permanently and specifically in the proper "niche" represents the Next Generation Gene Therapy, hopefully to be considered as the gold standard therapy in the near future.…”

Investigating Optimal Autologous Cellular Platforms for Prenatal or Perinatal Factor VIII Delivery to Treat Hemophilia A

“…Approximately, 75% of HA patients have a family history, and fetal DNA within maternal blood enables non-invasive HA diagnosis as early as 7 gestational weeks ( Tsui et al, 2011 ; Hudecova et al, 2017 ; Ragni, 2017 ; Camunas-Soler et al, 2018 ), making prenatal therapy possible ( Porada et al, 2014 ). Adding to the advantages of prenatal correction are studies showing that fetal exposure to foreign antigen induces lifelong tolerance that resists postnatal challenge ( Tran et al, 2001 ; Waddington et al, 2004 ; Colletti et al, 2008 ; Porada and Almeida-Porada, 2012 ; Porada et al, 2013 ; Peranteau et al, 2015 ; Takahashi et al, 2015 ; Davey et al, 2017 ). As such, even if not curative, prenatal treatment would permit postnatal therapies without fear of immune-related complications.…”