PMID- 15720961
OWN - NLM
STAT- MEDLINE
DCOM- 20060302
LR  - 20121115
IS  - 1541-0714 (Electronic)
IS  - 1540-3408 (Linking)
VI  - 4
IP  - 2
DP  - 2005 Mar
TI  - Stem cell transplantation and gene therapy for hemoglobinopathies.
PG  - 126-31
AB  - The b-thalassemias and sickle cell disorders are the most common genetic diseases 
      worldwide. Although improvements in conservative treatment have considerably 
      improved the prognosis of hemoglobinopathies, stem cell transplantation remains 
      the only cure for thalassemia and sickle cell disease. Results of transplants in 
      these diseases have steadily improved over the last two decades due to 
      improvements in preventive strategies, effective control of transplant-related 
      complications and development of new preparative regimens. High-resolution human 
      leukocyte antigen (HLA) typing has enabled physicians to perform transplant from 
      unrelated volunteer donors for thalassemia with results comparable with those 
      obtained employing an HLA-identical sibling. Current understandings of stable 
      mixed chimerism (MC) in patients with hemoglobinopathies provide a rationale for 
      the use of less intensive conditioning regimens and future gene therapy. Despite 
      recent advances in animal models, the clinical application of gene therapy for 
      hemoglobinopathies is unlikely to be a reality for at least near future. With the 
      advances in transplantation for thalassemia, all sickle cell disease patients 
      should be offered stem cell transplantation with an human leukocyte antigen 
      (HLA)-identical donor. This review focuses on the current status of stem cell 
      transplantation for hemoglobinopathies.
FAU - Gaziev, Javid
AU  - Gaziev J
AD  - Mediterranean Institute of Hematology, Rome, Italy.
FAU - Lucarelli, Guido
AU  - Lucarelli G
LA  - eng
PT  - Journal Article
PT  - Review
PL  - United States
TA  - Curr Hematol Rep
JT  - Current hematology reports
JID - 101151358
SB  - IM
MH  - *Genetic Therapy
MH  - *Hematopoietic Stem Cell Transplantation
MH  - Hemoglobinopathies/*therapy
MH  - Humans
RF  - 44
EDAT- 2005/02/22 09:00
MHDA- 2006/03/03 09:00
CRDT- 2005/02/22 09:00
PHST- 2005/02/22 09:00 [pubmed]
PHST- 2006/03/03 09:00 [medline]
PHST- 2005/02/22 09:00 [entrez]
PST - ppublish
SO  - Curr Hematol Rep. 2005 Mar;4(2):126-31.