NCL in Merino sheep is a subunit c-storing disease, clinically and pathologically similar to NCL in South Hampshire sheep. We propose that the disease in both breeds represents mutation at the same gene locus in chromosomal region OAR7q13-15.

Hepatic iron overload is commonly identified in hematopoietic stem cell transplant patients and can be accurately diagnosed by liver biopsy. In addition, hepatic iron overload has been identified in patients receiving as few as 25 units of packed red blood cells, with elevated posttransplant serum ferritin levels, and with blood stream infections.

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The mean number of infused CD34+ve cells was 6.7x 10 6 /kg recipient weight. All patients engrafted rapidly at a median rate of 15 days for neutrophils and 16 days for platelets. Although the total incidence of cGVHD was 43%, only 17% had extensive cGVHD, and the remaining 26% had the limited form which was very well controlled under a small-dose of immuno-suppressive therapy. By subgroup analysis, we found that the vast majority of patients with extensive cGVHD received the initial lower dose of Flu (75mg/ m2), and half of them were >30 years old. Since then, we've standardized the dose to be 120mg/m2. At a follow up period of up to 9 years (median 5.9 years), both DFS & OS were 79.2%. Conclusion: Allografting SAA patients with PBSCs, using the Flu-Cy regimen has a significantly lower cost, with excellent tolerability, and promising outcome regarding engraftment, DFS & OS. The incidence of cGVHD can be considered acceptable, putting in mind the higher patients' age and the initial lower dose of Flu that we've used. However, this approach definitely needs a larger cohort of patients to be randomly compared to the standard Cy-ATG regimen with the use of BM cells.