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A highly efficacious lymphocyte chemoattractant, stromal cell-derived factor 1 (SDF-1)
A highly efficacious lymphocytes chemotactic activity in the supernatants of the murine bone marrow stroma cell line MS-5 is identified which attracts 10-fold more lymphocytes in vitro than currently described lymphocyte chemoattractants. Expand
The Chemokine SDF-1 Is a Chemoattractant for Human CD34+ Hematopoietic Progenitor Cells and Provides a New Mechanism to Explain the Mobilization of CD34+ Progenitors to Peripheral Blood
Hematopoietic progenitor cells migrate in vitro and in vivo towards a gradient of the chemotactic factor stromal cell-derived factor-1 (SDF-1) produced by stromal cells. This is the firstExpand
Correction of ADA-SCID by Stem Cell Gene Therapy Combined with Nonmyeloablative Conditioning
Sustained engraftment of engineered HSCs with differentiation into multiple lineages resulted in increased lymphocyte counts, improved immune functions, and lower toxic metabolites, indicating the safety and efficacy of HSC gene therapy combined with nonmyeloablative conditioning for the treatment of SCID. Expand
Gene therapy for immunodeficiency due to adenosine deaminase deficiency.
Gene therapy, combined with reduced-intensity conditioning, is a safe and effective treatment for SCID in patients with ADA deficiency and effective protection against infections and improvement in physical development made a normal lifestyle possible. Expand
Autoantibodies against type I IFNs in patients with life-threatening COVID-19
A means by which individuals at highest risk of life-threatening COVID-19 can be identified is identified, and the hypothesis that neutralizing auto-Abs against type I IFNs may underlie critical CO VID-19 is tested. Expand
Lentiviral Hematopoietic Stem Cell Gene Therapy Benefits Metachromatic Leukodystrophy
The reconstitution of ARSA activity in the cerebrospinal fluid and the arrested progression of neurodegenerative disease in the three treated patients demonstrate that the transplanted cells, or their progeny, can seed the nervous system and deliver therapeutic levels of active enzyme. Expand
Recent advances in understanding the pathophysiology of Wiskott-Aldrich syndrome.
A full understanding of the mechanisms leading to defects in T, B, and dendritic cells still remains poorly understood is still needed to further implement new therapeutic strategies for this peculiar immunodeficiency. Expand
Lentiviral Hematopoietic Stem Cell Gene Therapy in Patients with Wiskott-Aldrich Syndrome
A clinical protocol based on lentiviral vector (LV) gene transfer into autologous hematopoietic stem/progenitor cells (HSCs) resulted in robust, stable, and long-term engraftment of gene-corrected HSCs in the patients’ bone marrow, and the findings support the use of LV gene therapy to treat patients with WAS. Expand
Comprehensive genomic access to vector integration in clinical gene therapy
A model to define genomic access to the viral integration site that provides optimal restriction motif combinations and minimizes the percentage of nonaccessible insertion loci is developed and a new nonrestrictive LAM-PCR approach is introduced. Expand
SAP controls the cytolytic activity of CD8+ T cells against EBV-infected cells.
The results demonstrate that the SAP/2B4 pathway plays a key role in CTL lytic activity against EBV-positive targets by promoting the polarization of the lytic machinery. Expand