Effects of enzyme replacement therapy on immune function in ADA deficiency patient

Yumiko Nakazawa; Toshinao Kawai; Toru Uchiyama; Fumihiro Goto; Nobuyuki Watanabe; Takanobu Maekawa; Akira Ishiguro; Torayuki Okuyama; Makoto Otsu; Masafumi Yamada; Michael S Hershfield; Tadashi Ariga; Masafumi Onodera

doi:10.1016/j.clim.2015.06.011

Effects of enzyme replacement therapy on immune function in ADA deficiency patient

Clin Immunol. 2015 Dec;161(2):391-3. doi: 10.1016/j.clim.2015.06.011. Epub 2015 Jun 27.

Affiliations

¹ Department of Human Genetics, National Research Institute for Child Health and Development, Japan.
² Department of General Pediatrics &Interdisciplinary Medicine, National Center for Child Health and Development, Japan.
³ Clinical Laboratory Medicine, National Center for Child Health and Development, Japan.
⁴ Center for Stem Cell Biology and Regenerative Medicine, Tokyo University, Japan.
⁵ Department of Pediatrics, Hokkaido University, Japan.
⁶ Department of Biochemistry, Duke University Medical Center, USA.
⁷ Department of Human Genetics, National Research Institute for Child Health and Development, Japan. Electronic address: onodera-m@ncchd.go.jp.

PMID: 26122173
DOI: 10.1016/j.clim.2015.06.011

No abstract available

Keywords: Adenosine deaminase deficiency; Enzyme replacement therapy; Immune reconstitution; PEG-ADA; Severe combined immunodeficiency; Stem cell gene therapy.

Publication types

Case Reports
Letter
Research Support, Non-U.S. Gov't

MeSH terms

Adenosine Deaminase / blood
Adenosine Deaminase / deficiency*
Adenosine Deaminase / immunology
Adenosine Deaminase / metabolism
Adenosine Deaminase / therapeutic use
Adolescent
Agammaglobulinemia / drug therapy*
Agammaglobulinemia / enzymology
Agammaglobulinemia / immunology
B-Lymphocytes / drug effects
B-Lymphocytes / immunology
B-Lymphocytes / metabolism
Enzyme Replacement Therapy / methods*
Female
Flow Cytometry
Humans
Lymphocyte Count
Severe Combined Immunodeficiency / drug therapy*
Severe Combined Immunodeficiency / enzymology
Severe Combined Immunodeficiency / immunology
T-Lymphocytes / drug effects
T-Lymphocytes / immunology
T-Lymphocytes / metabolism
Treatment Outcome

Substances

Adenosine Deaminase
pegademase bovine

Supplementary concepts

Severe combined immunodeficiency due to adenosine deaminase deficiency