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gene-modified autologous hematopoietic stem cells expressing ADA
- Development stage
- Unknown
- Lead developer
- GSK
- Modality
- Gene Therapies, Stem Cell Therapies → Cell Therapies
- Administration
- Intravenous
01
Overview
Gene-modified autologous hematopoietic stem cells expressing ADA is an ex vivo gene therapy being developed for the treatment of adenosine deaminase deficiency severe combined immunodeficiency (ADA-SCID). The therapy involves isolating autologous CD34+ hematopoietic stem and progenitor cells from the patient, transducing them ex vivo with a self-inactivating lentiviral vector carrying a functional human adenosine deaminase (ADA) gene, and reinfusing the modified cells back into the patient. Once engrafted, these cells differentiate into functional immune cells that express active ADA, thereby restoring purine metabolism, reducing toxic metabolite accumulation, and reconstituting the patient's immune system. Beijing Children's Hospital has evaluated this approach in clinical trials in China.
- Other names
- autologous ADA-expressing hematopoietic stem cellsautologous hematopoietic stem cell gene therapy expressing ADAgene-modified autologous hematopoietic stem cells expressing adenosine deaminase
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