Transduction efficiency of the infused product and number of transduced cells are variable from patient to patient. The lack of selective advantage for gene-marked ADA-expressing granulocytes results in a similar proportion of transduced granulocytes to that found in hematopoietic stem cells (HSCs). In contrast, gene-marked lymphocytes expressing ADA exhibit a strong selective advantage, resulting in a higher percent transduction and vector copy number (VCN) in lymphocytes compared with granulocytes. On the left, a low transduced HSC number results in lymphocytes bearing a limited number of unique integrants and persistent lymphopenia. On the right, high transduced HSC numbers translate to better correction of lymphopenia and polyclonal repertoire with higher number of unique integrants.

Transduction efficiency of the infused product and number of transduced cells are variable from patient to patient. The lack of selective advantage for gene-marked ADA-expressing granulocytes results in a similar proportion of transduced granulocytes to that found in hematopoietic stem cells (HSCs). In contrast, gene-marked lymphocytes expressing ADA exhibit a strong selective advantage, resulting in a higher percent transduction and vector copy number (VCN) in lymphocytes compared with granulocytes. On the left, a low transduced HSC number results in lymphocytes bearing a limited number of unique integrants and persistent lymphopenia. On the right, high transduced HSC numbers translate to better correction of lymphopenia and polyclonal repertoire with higher number of unique integrants.

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