Safer vectors for gene therapy of primary immunodeficiencies.
Curr Gene Ther
; 9(4): 291-305, 2009 Aug.
Article
em En
| MEDLINE
| ID: mdl-19534652
ABSTRACT
Primary immunodeficiencies (PID) are caused by mutations in genes that impair the development or activity of the immune system. Although bone marrow transplants achieve long time restoration in up to 90% of treated patients, morbidity and mortality are still high for some PID and adequate donors are not always available. Gene Therapy (GT) was envisioned as an alternative treatment for PID by inserting the correct gene into the patient's haematopoietic stem cells (HSCs). Up to date, GT for PID has succeeded in 40 of 44 patients treated in four clinical trials. However, five children enrolled in the SCID-X1 clinical trial developed leukaemia-like disease produced by aberrant expression of oncogenes. This phenomenon resulted fatal in one patient and represented a severe setback for gene therapy. Since then, vector development has been a priority in the GT field, by refining existing Murine laeukemia virus (MLV)-based vectors or by developing new ones. This review summarize existing methodologies for PID GT highlighting the importance of animal models in the PID GT success and focusing on new gene transfer vectors to achieve safe, efficient and stable gene modification.
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Coleções:
01-internacional
Temas:
Geral
/
Tratamento
Base de dados:
MEDLINE
Assunto principal:
Transfecção
/
Terapia Genética
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Vetores Genéticos
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Síndromes de Imunodeficiência
Limite:
Animals
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Humans
Idioma:
En
Revista:
Curr Gene Ther
Assunto da revista:
GENETICA MEDICA
/
TERAPEUTICA
Ano de publicação:
2009
Tipo de documento:
Article
País de afiliação:
Espanha