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PPT : Gene therapy for beta-thalassemia

Gene therapy trials in the world
* Gene transfer of a regulated ß-globin gene in HSCs would reduce the imbalance between a- and ß-globin chains in erythroid cells
* Transplantation of autologous, genetically corrected HSCs would represent an alternative therapy for thalassemic patients lacking a suitable bone marrow donor
Purification of CD34+ cells
TERAPIA GENICA DELLA ß-TALASSEMIA
Infusion of genetically-corrected cells
Gene therapy for ß-thalassemia
* efficient gene transfer into target cells
* adequate level of transgene expression
* persistence of gene expression
* regulation of gene expression
* tolerance to transgene product
The success of gene therapy is based on:
Gene therapy of ADA-SCID:from lab to clinic
Sadelain lab. (Nature 2000, Blood 2003, Blood 2007)
LV TNS9 and derivatives
Correction of murine thalassemia
Correction of human thalassemic cells
Correction of murine thalassemia
In vivo selection of corrected cells
Correction of human thalassemic cells
Lentiviral vectors in GT
Lentiviral vectors in GT:the French trial
Center of excellence for gene therapy
The success of gene therapy for ADA-SCID
HSR-TIGET
Pipeline of GT clinical trials
MLD(metachromatic leukodystrophy)
WAS (Wiskott-Aldrich syndrome)
ß-thalassemia
MPS I (mucopolysaccharidosis)
GLD (globoid leukodystrophy)
CGD (chronic granulomatosis disease)

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thalassemia

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