Cxcr4-haploinsufficient bone marrow transplantation corrects leukopenia in an unconditioned WHIM syndrome model
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DOI: 10.1172/jci120375
Abstract: For gene therapy of gain-of-function autosomal dominant diseases, either correcting or deleting the disease allele is potentially curative. To test whether there may be an advantage of one approach over the other for WHIM (warts,… read more here.
Keywords: unconditioned whim; whim syndrome; model; transplantation ... See more keywords