Efficacy of Gene Therapy for X-Linked Severe Combined Immunodeficiency
Hôpital Necker-Enfants Malades · Inserm · +10 more institutions
Abstract
The outcomes of gene therapy to correct congenital immunodeficiencies are unknown. We reviewed long-term outcomes after gene therapy in nine patients with X-linked severe combined immunodeficiency (SCID-X1), which is characterized by the absence of the cytokine receptor common gamma chain.
The nine patients, who lacked an HLA-identical donor, underwent ex vivo retrovirus-mediated transfer of gamma chain to autologous CD34+ bone marrow cells between 1999 and 2002. We assessed clinical events and immune function on long-term follow-up.
Citation impact
- FWCI
- 40.75
- Percentile
- 100%
- References
- 23
Authors
19- SHSalima Hacein‐Bey‐AbinaCorresponding
Hôpital Necker-Enfants Malades, Inserm, Université Paris Cité, Institut Necker Enfants Malades, Assistance Publique – Hôpitaux de Paris
- JHJulia Hauer
Inserm, Institut Necker Enfants Malades
- ALAnnick Lim
Inserm, Institut Pasteur
- CPCapucine Pïcard
Université Paris Cité, Centre de Référence Déficits Immunitaires Héréditaires
- GPGary P. Wang
University of Pennsylvania
Topics & keywords
- Medicine
- Severe combined immunodeficiency
- Genetic enhancement
- Immunodeficiency
- Hematopoietic stem cell transplantation
- Immunology
- Immune system
- Transplantation
- Good health and well-being