Details

Autor(en) / Beteiligte

• Zhang, L
• Thrasher, A J
• Gaspar, H B

Titel

Current progress on gene therapy for primary immunodeficiencies

Ist Teil von

• Gene therapy, 2013-10, Vol.20 (10), p.963-969

Ort / Verlag

England: Nature Publishing Group

Erscheinungsjahr

2013

Quelle

MEDLINE

Beschreibungen/Notizen

• Primary immunodeficiencies have played a major role in the development of gene therapy for monogenic diseases of the bone marrow. The last decade has seen convincing evidence of long-term disease correction as a result of ex vivo viral vector-mediated gene transfer into autologous haematopoietic stem cells. The success of these early studies has been balanced by the development of vector-related insertional mutagenic events. More recently the use of alternative vector designs with self-inactivating designs, which have an improved safety profile has led to the initiation of a wave of new studies that are showing early signs of efficacy. The ongoing development of safer vector platforms and gene-correction technologies together with improvements in cell-transduction techniques and optimised conditioning regimes is likely to make gene therapy amenable for a greater number of PIDs. If long-term efficacy and safety are shown, gene therapy will become a standard treatment option for specific forms of PID.