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Successful Peripheral T-Lymphocyte-Directed Gene Transfer for a
Patient With Severe Combined Immune Deficiency Caused by Adenosine
Deaminase Deficiency
Masafumi Onodera,
Tadashi Ariga,
Nobuaki Kawamura,
Ichiro Kobayashi,
Makoto Ohtsu,
Masafumi Yamada,
Atsushi Tame,
Hirofumi Furuta,
Motohiko Okano,
Shuzo Matsumoto,
Hitoshi Kotani,
Gerard J. McGarrity,
R. Michael Blaese, and
Yukio Sakiyama
From the Department of Pediatrics, Hokkaido University School of
Medicine, Sapporo, Japan; Clinical Gene Therapy Branch, National Human
Genome Research Institute, National Institutes of Health, Bethesda, MD;
Institute of Medical Science, Health Sciences University of Hokkaido,
Ainosato, Sapporo, Japan; and Genetic Therapy, Inc, Gaithersburg, MD.
Ten patients with adenosine deaminase deficiency
(ADA ) have been enrolled in gene therapy
clinical trials since the first patient was treated in September 1990. We describe a Japanese ADA severe combined immune
deficiency (SCID) patient who has received periodic infusions of
genetically modified autologous T lymphocytes transduced with the human
ADA cDNA containing retroviral vector LASN. The percentage
of peripheral blood lymphocytes carrying the transduced ADA gene has
remained stable at 10% to 20% during the 12 months since the fourth
infusion. ADA enzyme activity in the patient's circulating T cells,
which was only marginally detected before gene transfer, increased to
levels comparable to those of a heterozygous carrier individual and was
associated with increased T-lymphocyte counts and improvement of the
patient's immune function. The results obtained in this trial are in
agreement with previously published observations and support the
usefulness of T lymphocyte-directed gene transfer in the treatment of
ADASCID.
Blood, Vol. 91 No. 1 (January 1), 1998:
pp. 30-36
© 1998 by The American Society of Hematology.
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