Gene Therapy Communication: Use of a Needs Assessment to Drive Decision-AIDS for Gene Therapy for Rare Diseases (GENETX)

Eligibility Criteria

• 1. For Group 1 participants only (Undergone Gene Therapy):
• * Parent/caregiver whose child has undergone gene therapy. OR Young adult aged between 18 years to 35 years who has undergone gene therapy.
• * Willingness to participate in one-on-one video interview with a study team member using a personal mobile device or computer with working internet connection.
• * Must be willing to provide verbal informed consent.
• * Release of information form signed by participant providing our study team with permission to contact healthcare provider to verify their diagnosis and receipt of gene therapy (if received).
• * Successful verification of diagnosis of rare genetic disease targeted for treatment using gene therapy.
• * A positive confirmation on receipt of gene therapy and type received from their healthcare provider (only for those received gene therapy).
• 2. For Group 2 participants only (Offered, but did not Undergo Gene Therapy):
• * Parent/caregiver of children (or young adults aged between 18 years to 35 years) with a rare genetic disease who had been offered but were not eligible for a trial or decided against receiving gene therapy.
• * Willingness to participate in one-on-one video interview with a study team member using a personal mobile device or computer with working internet connection.
• * Must be willing to provide verbal informed consent.
• * Signed release of information form providing GeneTx study team with permission to contact participant's healthcare provider to verify the diagnosis.
• * Successful verification of diagnosis of rare genetic disease targeted for treatment using gene therapy.
• 3. For Group 3 participants only (Provider Interviews):
• * Healthcare worker who has provided care to ≥ 2 patients receiving gene therapy.
• * Willingness to participate in one-on-one video (or in-person) interview with a study team member using a personal mobile device or computer with working internet connection.
• * Informed consent from a study participant.
• * Participants who are unable to converse fluently in English will be excluded.
• * Inability or unwillingness of research participant to give verbal informed consent.
• * Participants who lack access to a computer or mobile device that supports video communications will be excluded.
• * Condition or chronic illness, which in the opinion of the PI/Co-I, makes participation unsafe or untenable (i.e., cognitive impairment, concurrent acute morbidity).