Survey of Health Care Provider Understanding of Gene Therapy Research for Inherited Metabolic Disorders

Joyanna Hansen, Radha Ramachandran, Jerry Vockley

Research output: Contribution to journalArticlepeer-review

3 Scopus citations


Purpose: The treatment of inherited metabolic disorders (IMDs) has traditionally relied on dietary interventions that are difficult to maintain, expensive, and socially isolating. The development of gene therapy for IMDs aims to provide sufficient gene activity to address the underlying causes of these conditions. This study surveyed health care providers (HCPs) to characterize their familiarity with gene therapy technologies and to identify educational needs across roles in a multidisciplinary care team. Methods: The link to a Web-based, 26-question survey was distributed to HCPs in North America and Europe who were involved in IMD patient care. Results were analyzed using descriptive statistics. Findings: Of the 590 survey link recipients, 64 completed the survey. Of these, 35 (55%) respondents were physicians, 23 (36%) were dietitians, 3 (5%) were nurse practitioners, 2 (3%) were genetic counselors, and 1 (2%) was a nurse. Most survey respondents (88% [n = 56 of 64]) reported the belief that gene therapy for IMDs would be available within 5 years of study conduct. Although nearly all physicians (97% [n = 34 of 35]) expressed awareness of gene therapy, rates of reported familiarity were lower among dietitians (57% [n = 13 of 23]); confidence in conversations with colleagues and patients/caregivers was also discordant. Nearly all HCPs wanted education on gene therapy advancements, and the most preferred informational sources were published literature and congress presentations. Implications: There is an urgent need for education on topics related to gene therapy modalities. Professional education on gene therapies is desired across all specialties and will be important for unified treatment practices in IMD care.

Original languageEnglish (US)
Pages (from-to)1045-1056
Number of pages12
JournalClinical therapeutics
Issue number8
StatePublished - Aug 2022


  • gene therapy
  • gene transfer
  • inborn errors of metabolism
  • inherited metabolic disorders
  • phenylketonuria
  • survey

ASJC Scopus subject areas

  • Pharmacology
  • Pharmacology (medical)


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