PMID- 15687233
OWN - NLM
STAT- MEDLINE
DCOM- 20050712
LR  - 20210206
IS  - 0006-4971 (Print)
IS  - 0006-4971 (Linking)
VI  - 105
IP  - 11
DP  - 2005 Jun 1
TI  - Failure of SCID-X1 gene therapy in older patients.
PG  - 4255-7
AB  - Gene therapy has been shown to be a highly effective treatment for infants with 
      typical X-linked severe combined immunodeficiency (SCID-X1, gammac-deficiency). 
      For patients in whom previous allogeneic transplantation has failed, and others 
      with attenuated disease who may present later in life, the optimal treatment 
      strategy in the absence of human leukocyte antigen (HLA)-matched donors is 
      unclear. Here we report the failure of gene therapy in 2 such patients, despite 
      effective gene transfer to bone marrow CD34(+) cells, suggesting that there are 
      intrinsic host-dependent restrictions to efficacy. In particular, there is likely 
      to be a limitation to initiation of normal thymopoiesis, and we therefore suggest 
      that intervention for these patients should be considered as early as possible.
FAU - Thrasher, Adrian J
AU  - Thrasher AJ
AD  - Molecular Immunology Unit, Institute of Child Health, London, United Kingdom.
FAU - Hacein-Bey-Abina, Salima
AU  - Hacein-Bey-Abina S
FAU - Gaspar, H Bobby
AU  - Gaspar HB
FAU - Blanche, Stephane
AU  - Blanche S
FAU - Davies, E Graham
AU  - Davies EG
FAU - Parsley, Kathryn
AU  - Parsley K
FAU - Gilmour, Kimberly
AU  - Gilmour K
FAU - King, Douglas
AU  - King D
FAU - Howe, Steven
AU  - Howe S
FAU - Sinclair, Joanna
AU  - Sinclair J
FAU - Hue, Christophe
AU  - Hue C
FAU - Carlier, Frederique
AU  - Carlier F
FAU - von Kalle, Christof
AU  - von Kalle C
FAU - de Saint Basile, Genevieve
AU  - de Saint Basile G
FAU - le Deist, Francoise
AU  - le Deist F
FAU - Fischer, Alain
AU  - Fischer A
FAU - Cavazzana-Calvo, Marina
AU  - Cavazzana-Calvo M
LA  - eng
GR  - Wellcome Trust/United Kingdom
PT  - Case Reports
PT  - Journal Article
PT  - Research Support, Non-U.S. Gov't
DEP - 20050201
PL  - United States
TA  - Blood
JT  - Blood
JID - 7603509
RN  - 0 (Antigens, CD34)
SB  - IM
MH  - Adult
MH  - Antigens, CD34
MH  - Bone Marrow Cells/metabolism
MH  - Bone Marrow Transplantation
MH  - Genetic Diseases, X-Linked/*therapy
MH  - *Genetic Therapy
MH  - Humans
MH  - Male
MH  - Severe Combined Immunodeficiency/*therapy
MH  - Transduction, Genetic
MH  - Treatment Failure
EDAT- 2005/02/03 09:00
MHDA- 2005/07/13 09:00
CRDT- 2005/02/03 09:00
PHST- 2005/02/03 09:00 [pubmed]
PHST- 2005/07/13 09:00 [medline]
PHST- 2005/02/03 09:00 [entrez]
AID - S0006-4971(20)53462-9 [pii]
AID - 10.1182/blood-2004-12-4837 [doi]
PST - ppublish
SO  - Blood. 2005 Jun 1;105(11):4255-7. doi: 10.1182/blood-2004-12-4837. Epub 2005 Feb 
      1.