Gene Therapy: Knowledge, Attitudes, and Preferences Among Individuals with Alpha-1 Antitrypsin Deficiency
Alpha-1 antitrypsin patients show strong willingness for gene therapy but knowledge gaps exist—better education could boost trial enrollment and treatment uptake.
Over 1100 AATD patients surveyed show moderate gene therapy knowledge gaps but generally positive attitudes, with higher knowledge strongly predicting willingness to participate in trials and accept FDA-approved therapy — a finding with direct implications for gene therapy trial recruitment strategies in this rare disease. Patient preference for dual liver/lung targeting provides actionable guidance for gene therapy vector design priorities.
What the study was
- Study design
- Cross-sectional electronic survey
- Population
- Alpha-1 antitrypsin deficiency (AATD) patient community (AlphaNet); n=1112
- Sample size
- 1112
- Category
- Public Health
- Maturity
- Exploratory
- Journal
- Chronic Obstructive Pulmonary Diseases
Why it surfaced
First gene therapy attitude survey in AATD community; directly useful for trial design and patient engagement in an orphan disease; n=1112 is substantial for a rare disease survey.
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