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Beliefs and Values About Gene Therapy and In-Utero Gene Editing in Patients with Hemophilia and Their Relatives.

Tania C Vasquez-LoarteTiffany Lin LucasJulie Harris-WaiDeborah J Bowen
Published in: The patient (2021)
Patients with severe hemophilia perceive gene therapy as a potential cure, while gene editing was more controversial. Patients still have questions that remain to be answered regarding safety and efficacy that should be assessed with long-term follow up studies.
Keyphrases
  • gene therapy
  • end stage renal disease
  • ejection fraction
  • newly diagnosed
  • chronic kidney disease
  • early onset
  • childhood cancer