- Conditions
- Wiskott-Aldrich Syndrome
- Interventions
- Retrovirus-mediated gene transfer
- Biological
- Lead sponsor
- David Williams
- Other
- Eligibility
- 3 Months to 35 Years · Male only
- Enrollment
- 5 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2011 – 2024
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
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Showing 1–6
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- Conditions
- Sickle Cell Disease
- Interventions
- single infusion of autologous bone marrow derived CD34+ HSC cells transduced with the lentiviral vector containing a short-hairpin RNA targeting BCL11a
- Biological
- Lead sponsor
- David Williams
- Other
- Eligibility
- 3 Years to 40 Years
- Enrollment
- 10 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2026
- U.S. locations
- 2
- States / cities
- Los Angeles, California • Boston, Massachusetts
- Conditions
- Sickle Cell Disease
- Interventions
- Autologous CD34+ HSC cells transduced with the lentiviral vector containing a shRNA targeting BCL11a
- Biological
- Lead sponsor
- David Williams
- Other
- Eligibility
- 13 Years to 55 Years
- U.S. locations
- 1
- States / cities
- Boston, Massachusetts
- Conditions
- Sickle Cell Disease
- Interventions
- Autologous CD34+ HSC cells transduced with the lentiviral vector containing a shRNA targeting BCL11a
- Biological
- Lead sponsor
- David Williams
- Other
- Eligibility
- 13 Years to 40 Years
- Enrollment
- 25 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2022 – 2027
- U.S. locations
- 9
- States / cities
- Los Angeles, California • Oakland, California • Sacramento, California + 4 more
- Conditions
- Severe Combined Immunodeficiency, X Linked, Gene Therapy
- Interventions
- autologous CD34+ cell transduced with G2SCID vector
- Biological
- Lead sponsor
- David Williams
- Other
- Eligibility
- 0 Years to 5 Years · Male only
- Enrollment
- 12 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2018 – 2028
- U.S. locations
- 4
- States / cities
- Los Angeles, California • Atlanta, Georgia • Boston, Massachusetts + 1 more
- Conditions
- Severe Combined Immunodeficiency
- Interventions
- Gene transfer
- Biological
- Lead sponsor
- David Williams
- Other
- Eligibility
- Male only
- Enrollment
- 8 participants
- Healthy volunteers
- Healthy volunteers not accepted
- Timeline
- 2010 – 2023
- U.S. locations
- 3
- States / cities
- Los Angeles, California • Boston, Massachusetts • Cincinnati, Ohio