Clinical Research Directory

Inclusion Criteria: 1. For Group 1 participants only (Undergone Gene Therapy): * Parent/caregiver whose child has undergone gene therapy. OR Parent/caregiver of a child who died after receiving gene therapy at least 6 months prior to enrollment, but no more than 24 months prior to enrollment, to be contacted no sooner than 3 months after the death has occurred and no longer than 2 years. OR Patients age 8 and above who have 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 patients 8 and above ) 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. 4. For Group 4 participants only (Undergone Gene Therapy for Bone Marrow Failure Condition): * Parent/caregiver whose child has undergone gene therapy. OR Parent/caregiver of a child who died after receiving gene therapy at least 6 months prior to enrollment, but no more than 24 months prior to enrollment, to be contacted no sooner than 3 months after the death has occurred and no longer than 2 years. OR Patients age 8 and above who have 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). 5. For Group 5 participants only (Offered, but did not Undergo Gene Therapy for Bone Marrow Failure Condition ): * Parent/caregiver of children (or patients 8 and above ) with a bone marrow failure 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. 6. For Group 6 participants only (Never offered gene therapy for Bone Marrow Failure Condition): * Parent/caregiver of children (or patients 8 and above ) with a bone marrow failure disease who had not been offered 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. 7. For Group 7 participants only (Provider Interviews for Bone Marrow Failure Condition): * 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. Exclusion Criteria (for all 7 groups): * 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).