Induced pluripotent stem cells and severe combined immunodeficiency: merely disease modeling or potentially a novel cure?
Pediatr Res
; 71(4 Pt 2): 427-32, 2012 Apr.
Article
em En
| MEDLINE
| ID: mdl-22430378
ABSTRACT
For most, but not all, types of severe combined immunodeficiency (SCID) the underlying molecular defects are known, in principle allowing the cure of affected children via gene therapy. Typically such approaches have used autologous hematopoietic stem cells modified to express a therapeutic gene via γ-retroviral vectors. Insertional mutagenesis has emerged as a significant risk for successful application of this type of gene therapy. Therefore, lentiviral vectors with a self-inactivating design have been developed. Recent advances in stem cell technology using induced pluripotent stem cells (iPSCs) allow an entire different approach to gene therapy for SCID and other genetic disorders, namely by correction of the affected gene in patient-specific iPSCs followed by hematopoietic differentiation. Here, we review these recent advances in the field from an efficacy and safety point of view.
Texto completo:
1
Base de dados:
MEDLINE
Assunto principal:
Pediatria
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Terapia Genética
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Imunodeficiência Combinada Severa
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Transplante de Células-Tronco
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Células-Tronco Pluripotentes Induzidas
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Modelos Biológicos
Limite:
Child
/
Humans
Idioma:
En
Revista:
Pediatr Res
Ano de publicação:
2012
Tipo de documento:
Article
País de afiliação:
Holanda