PMID- 19776764
OWN - NLM
STAT- MEDLINE
DCOM- 20100629
LR  - 20121115
IS  - 1476-5462 (Electronic)
IS  - 0969-7128 (Linking)
VI  - 16
IP  - 11
DP  - 2009 Nov
TI  - Progress and prospects: gene therapy for inherited immunodeficiencies.
PG  - 1285-91
LID - 10.1038/gt.2009.127 [doi]
AB  - Haematopoietic stem cell transplantation (HSCT) is now widely used to treat 
      primary immunodeficiencies (PID). For patients with specific disorders (severe 
      combined immunodeficiency (SCID)-X1, adenosine deaminase deficiency (ADA)-SCID, 
      X-chronic granulomatous disease (CGD) and Wiskott-Aldrich Syndrome (WAS)) who 
      lack a suitable human leukocyte antigen (HLA)-matched donor, gene therapy has 
      offered an important alternative treatment option. The success of gene therapy 
      can be attributed, in part, to the selective advantage offered to gene-corrected 
      cells, the avoidance of graft-versus-host disease and to the use of 
      pre-conditioning in patients with chemotherapy to facilitate engraftment of 
      corrected cells. Adverse events have been encountered and this has led to 
      detailed characterization of retroviral vector integration profiles. A new 
      generation of self-inactivating retroviral and lentiviral vectors have been 
      designed to address these safety concerns, and are at an advanced stage of 
      preparation for the next phase of clinical testing.
FAU - Qasim, W
AU  - Qasim W
AD  - Molecular Immunology Unit, UCL Institute of Child Health, London, UK.
FAU - Gaspar, H B
AU  - Gaspar HB
FAU - Thrasher, A J
AU  - Thrasher AJ
LA  - eng
PT  - Journal Article
PT  - Review
DEP - 20090924
PL  - England
TA  - Gene Ther
JT  - Gene therapy
JID - 9421525
SB  - IM
MH  - Animals
MH  - Gene Silencing
MH  - Genetic Therapy/*methods/trends
MH  - Humans
MH  - Immunologic Deficiency Syndromes/*therapy
MH  - Male
MH  - Mutagenesis, Insertional
MH  - Severe Combined Immunodeficiency/therapy
MH  - Wiskott-Aldrich Syndrome/therapy
RF  - 28
EDAT- 2009/09/25 06:00
MHDA- 2010/06/30 06:00
CRDT- 2009/09/25 06:00
PHST- 2009/09/25 06:00 [entrez]
PHST- 2009/09/25 06:00 [pubmed]
PHST- 2010/06/30 06:00 [medline]
AID - gt2009127 [pii]
AID - 10.1038/gt.2009.127 [doi]
PST - ppublish
SO  - Gene Ther. 2009 Nov;16(11):1285-91. doi: 10.1038/gt.2009.127. Epub 2009 Sep 24.