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IL-3 or IL-7 increases ex vivo gene transfer efficiency in ADA-SCID BM CD34(+) cells while maintaining in vivo lymphoid potential
IL-3 or IL-7 increases ex vivo gene transfer efficiency in ADA-SCID BM CD34(+) cells while maintaining in vivo lymphoid potential MOLECULAR THERAPY Ficara, F., Superchi, D. B., Hernandez, R. J., Mocchetti, C., Carballido-Perrig, N., Andolfi, G., Deola, S., Colombo, A., Bordignon, C., Carballido, J. M., Roncarolo, M. G., Aiuti, A. 2004; 10 (6): 1096-1108Abstract
To improve maintenance and gene transfer of human lymphoid progenitors for clinical use in gene therapy of adenosine deaminase (ADA)-deficient SCID we investigated several gene transfer protocols using various stem cell-enriched sources. The lymphoid differentiation potential was measured by an in vitro clonal assay for B/NK cells and in the in vivo SCID-hu mouse model. Ex vivo culture with the cytokines TPO, FLT3-ligand, and SCF (T/F/S) plus IL-3 or IL-7 substantially increased the yield of transduced bone marrow (BM) CD34(+) cells purified from ADA-SCID patients or healthy donors, compared to T/F/S alone. Moreover, the use of IL-3 or IL-7 significantly improved the maintenance of in vitro B cell progenitors from ADA-SCID BM cells and allowed the efficient transduction of B and NK cell progenitors. Under these optimized conditions transduced CD34(+) cells were efficiently engrafted into SCID-hu mice and gave rise to B and T cell progeny, demonstrating the maintenance of in vivo lymphoid reconstitution capacity. The protocol based on the T/F/S + IL-3 combination was included in a gene therapy clinical trial for ADA-SCID, resulting in long-term engraftment of stem/progenitor cells. Remarkably, gene-corrected BM CD34(+) cells obtained from one patient 4 and 11 months after gene therapy were capable of repopulating the lymphoid compartment of SCID-hu hosts.
View details for DOI 10.1016/j.ymthe.2004.08.014
View details for Web of Science ID 000226001000014
View details for PubMedID 15564141