Morbidity in an adenosine deaminase-deficient patient during 27 years of enzyme replacement therapy

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作者： Grunebaum, Eyal ^[1] ; Reid, Brenda ^[2] ; Naqvi, Ahmed ^[3] ; Hershfield, Michael S. ^[4] ; Kim, Vy Hong-Diep ^[5] ; Muller, Matthew P. ; Hicks, Lisa K. ; Lee, Erika ; Betschel, Stephen ; Roifman, Chaim M.

作者单位： St Michaels Hosp, Div Infect Dis, Toronto, ON, Canada ^[1] St Michaels Hosp, Div Hematol, Toronto, ON, Canada ^[2] Hosp Sick Children, Dept Pediat, Div Immunol & Allergy, Toronto, ON, Canada ^[3] Univ Toronto, Toronto, ON, Canada ^[4] Duke Univ, Div Med & Biochem, Med Ctr, Durham, NC USA ^[5]

关键词 Adenosine deaminase deficiency Severe combined immunodeficiency Lymphoma Gene therapy PEG Enzyme replacement therapy Mycobacterium genavense Long-term

发布时间 2020-10-12

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