New perspectives in gene therapy for inherited disorders

Research output: Contribution to journalArticlepeer-review


Gene therapy has become promising in many fields of medicine, as a single treatment could allow long-lasting and curative benefits. New medicines based on cell gene correction are expected to occur in upcoming years and will be hopefully part of the therapeutic armamentarium for inherited and acquired diseases. Issues related to the costs of these new therapies and access to care for all patients, and procedures and expertise needed to facilitate their application worldwide require to be addressed, together with long-term safety and efficacy monitoring.

Original languageEnglish
Pages (from-to)5-7
Number of pages3
JournalPediatric Allergy and Immunology
Issue numberS24
Publication statusPublished - Feb 1 2020


  • cancer
  • gene therapy
  • immunodeficiency
  • inherited disorders

ASJC Scopus subject areas

  • Pediatrics, Perinatology, and Child Health
  • Immunology and Allergy
  • Immunology


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