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Editorial
. 2021 Oct 14;138(15):1287-1288.
doi: 10.1182/blood.2021012300.

Built to last: gene therapy for ADA SCID

Sung-Yun Pai  1
Affiliations
Editorial

Built to last: gene therapy for ADA SCID

Sung-Yun Pai. Blood. .
No abstract available

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Conflict of interest statement

Conflict-of-interest disclosure: The author declares no competing financial interests. This work was supported by funding from the Intramural Research Program, National Institutes of Health, National Cancer Institute, Center for Cancer Research.

Figures

None
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.
See this image and copyright information in PMC

Comment on

  • Long-term outcomes after gene therapy for adenosine deaminase severe combined immune deficiency.
    Reinhardt B, Habib O, Shaw KL, Garabedian E, Carbonaro-Sarracino DA, Terrazas D, Fernandez BC, De Oliveira S, Moore TB, Ikeda AK, Engel BC, Podsakoff GM, Hollis RP, Fernandes A, Jackson C, Shupien S, Mishra S, Davila A, Mottahedeh J, Vitomirov A, Meng W, Rosenfeld AM, Roche AM, Hokama P, Reddy S, Everett J, Wang X, Luning Prak ET, Cornetta K, Hershfield MS, Sokolic R, De Ravin SS, Malech HL, Bushman FD, Candotti F, Kohn DB. Reinhardt B, et al. Blood. 2021 Oct 14;138(15):1304-1316. doi: 10.1182/blood.2020010260. Blood. 2021. PMID: 33974038 Free PMC article. Clinical Trial.

References

    1. Reinhardt B, Habib O, Shaw KL, et al. . Long-term outcomes after gene therapy for adenosine deaminase severe combined immune deficiency (ADA SCID). Blood. 2021;138(16):1304-1316. - PMC - PubMed
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    1. Kohn LA, Kohn DB.. Gene therapies for primary immune deficiencies. Front Immunol. 2021;12:648951. - PMC - PubMed

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