Outcomes in two Japanese adenosine deaminase-deficiency patients treated by stem cell gene therapy with no cytoreductive conditioning.

doi:10.1007/s10875-015-0157-1

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Article

Outcomes in two Japanese adenosine deaminase-deficiency patients treated by stem cell gene therapy with no cytoreductive conditioning.

Journal of clinical immunology - 1 May 2015

Otsu Makoto, Yamada Masafumi, Nakajima Satoru, Kida Miyuki, Maeyama Yoshihiro, Hatano Norikazu, Toita Nariaki, Takezaki Shunichiro, Okura Yuka, Kobayashi Ryoji, Matsumoto Yoshinori, Tatsuzawa Osamu, Tsuchida Fumiko, Kato Shunichi, Kitagawa Masanari, Mineno Junichi, Hershfield Michael S, Bali Pawan, Candotti Fabio, Onodera Masafumi, Kawamura Nobuaki, Sakiyama Yukio, Ariga Tadashi

Abstract excerpt

OBJECTIVE: We here describe treatment outcomes in two adenosine deaminase (ADA)-deficiency patients (pt) who received stem cell gene therapy (SCGT) with no cytoreductive conditioning. As this protocol has features distinct from those of other clinical trials, its results provide insights into SCGT for ADA deficiency. PATIENTS AND METHODS: Pt 1 was treated at age 4.7 years, whereas pt 2, who had previously...

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PMID: 25875699
DOI: 10.1007/s10875-015-0157-1

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