UNIV ADA-SCID的三种治疗手段 52 ERT 注射ADA蛋白 HSCT 骨髓移植 " direct administration of PEg-ADa Haemapoietic stem cell CONS transplantation PROS immune function within.Not curative detoxification ditioning, underlying Might not be availa ble in all man festations Autoimmune GENE THERAPY PROS CONS "transfer of normal Improvements yet to b 基因治疗 ADA gene into Unknown long-term safety autologous hScs" manifestation FIGURE 2 Potential treatment options for ADA deficiency include enzyme replacement therapy(ERT), hematopoietic stem cell transplantation (HSCT), and gene therapy(GT). There are varying benefits and potential problems for each available treat decide on the appropniate course of action. This table has Front Immuno 2016.7: 31412 ADA-SCID的三种治疗手段 Front. Immunol. 2016. 7:314 注射ADA蛋白 骨髓移植 基因治疗