PMID- 9475605
OWN - NLM
STAT- MEDLINE
DCOM- 19980324
LR  - 20121115
IS  - 0148-7299 (Print)
IS  - 0148-7299 (Linking)
VI  - 75
IP  - 3
DP  - 1998 Jan 23
TI  - Visible integration of the adenosine deaminase (ADA) gene into the recipient 
      genome after gene therapy.
PG  - 314-7
AB  - Gene therapy for patients with adenosine deaminase (ADA) deficiency has become 
      practical in the 1990s, and the exogenous gene has been reported to survive for 
      several years in the recipient genome. To evaluate the integration efficiency of 
      the ADA gene (ADA) into peripheral blood lymphocytes (PBL) of a patient with ADA 
      deficiency who is receiving gene therapy, we performed two-color interphase 
      fluorescence in situ hybridization (FISH) analysis by using digoxigenin-labeled 
      ADA-cDNA and the biotin-labeled lambda-genomic ADA clone as probes. After each of 
      9 sequential series of gene therapy, interphase nuclei of 100 mononuclear cells 
      from the patient were analyzed, and those of a LASN-producing cell line were used 
      as a control. FISH signals were detected with rhodamine and FITC for the cDNA and 
      the genomic DNA, respectively. The number of PBL giving a transgene signal grew 
      after the sequential gene therapies, and the proportion of signal-positive cells 
      reached about 10%. Our results indicate that the two-color FISH system can be 
      used as a potential aid to monitor the efficiency of the ADA gene therapy.
FAU - Egashira, M
AU  - Egashira M
AD  - Department of Human Genetics, Nagasaki University School of Medicine, Japan. 
      f0932@nagasaki-u.ac.jp
FAU - Ariga, T
AU  - Ariga T
FAU - Kawamura, N
AU  - Kawamura N
FAU - Miyoshi, O
AU  - Miyoshi O
FAU - Niikawa, N
AU  - Niikawa N
FAU - Sakiyama, Y
AU  - Sakiyama Y
LA  - eng
PT  - Case Reports
PT  - Journal Article
PT  - Research Support, Non-U.S. Gov't
PL  - United States
TA  - Am J Med Genet
JT  - American journal of medical genetics
JID - 7708900
RN  - EC 3.5.4.4 (Adenosine Deaminase)
SB  - IM
MH  - 3T3 Cells
MH  - Adenosine Deaminase/blood/*genetics
MH  - Animals
MH  - Child, Preschool
MH  - Genetic Therapy/*methods
MH  - Genome, Human
MH  - Humans
MH  - In Situ Hybridization, Fluorescence
MH  - Interphase/genetics
MH  - Male
MH  - Mice
MH  - Severe Combined Immunodeficiency/*genetics/*therapy
MH  - Transgenes/genetics
EDAT- 1998/02/25 03:05
MHDA- 2000/06/20 09:00
CRDT- 1998/02/25 03:05
PHST- 1998/02/25 03:05 [pubmed]
PHST- 2000/06/20 09:00 [medline]
PHST- 1998/02/25 03:05 [entrez]
AID - 10.1002/(SICI)1096-8628(19980123)75:3<314::AID-AJMG18>3.0.CO;2-S [pii]
PST - ppublish
SO  - Am J Med Genet. 1998 Jan 23;75(3):314-7.