2007/06/01 by Marina Cavazzana, Alain Fischer · 4 citations
Biochemistry, Genetics and Molecular Biology · Medicine · #Virus-based gene therapy research #RNA Interference and Gene Delivery #CAR-T cell therapy research
paper · pdf · doi:10.1172/jci30953
openalex publication_date 2007/06/01 · openalex created_date 2025/10/10 · openalex updated_date 2026/07/31
Inherited and acquired diseases of the hematopoietic system can be cured by allogeneic hematopoietic stem cell transplantation. This treatment strategy is highly successful when an HLA-matched sibling donor is available, but if not, few therapeutic options exist. Gene-modified, autologous bone marrow transplantation can circumvent the severe immunological complications that occur when a related HLA-mismatched donor is used and thus represents an attractive alternative. In this review, we summarize the advantages and limitations associated with the use of gene therapy to cure SCID. Insertional mutagenesis and technological improvements aimed at increasing the safety of this strategy are also discussed.