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Transfusion independence and HMGA2 activation after gene therapy of human β-thalassaemia
The β-haemoglobinopathies are the most prevalent inherited disorders worldwide. Gene therapy of β-thalassaemia is particularly challenging given the requirement for massive haemoglobin production inExpand
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Transplants of umbilical-cord blood or bone marrow from unrelated donors in adults with acute leukemia.
BACKGROUND Promising results of cord-blood transplants from unrelated donors have been reported in adults. METHODS We compared outcomes in 682 adults with acute leukemia who received aExpand
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Fanconi anemia is associated with a defect in the BRCA2 partner PALB2
The Fanconi anemia and BRCA networks are considered interconnected, as BRCA2 gene defects have been discovered in individuals with Fanconi anemia subtype D1. Here we show that a defect in theExpand
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Hematopoietic reconstitution in a patient with Fanconi's anemia by means of umbilical-cord blood from an HLA-identical sibling.
THE clinical manifestations of Fanconi's anemia, an autosomal recessive disorder, include progressive pancytopenia, a predisposition to neoplasia, and nonhematopoietic developmental anomalies.1 2 3...
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Comparison of outcomes of unrelated bone marrow and umbilical cord blood transplants in children with acute leukemia.
In order to compare the outcomes of unrelated umbilical cord blood transplants (UCBTs) or bone marrow transplants, 541 children with acute leukemia (AL) transplanted with umbilical cord blood (n =Expand
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Results of allogeneic bone marrow transplants for leukemia using donors other than HLA-identical siblings.
PURPOSE To compare outcomes of bone marrow transplants for leukemia from HLA-identical siblings, haploidentical HLA-mismatched relatives, and HLA-matched and mismatched unrelated donors. PATIENTS AExpand
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Imatinib mesylate discontinuation in patients with chronic myelogenous leukemia in complete molecular remission for more than 2 years.
In the present study, we address the issue of the discontinuation of imatinib mesylate (Gleevec) in chronic myelogenous leukemia with undetectable residual disease for more than 2 years. TwelveExpand
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Outcome of cord-blood transplantation from related and unrelated donors
Background Cord-blood banks have increased the use of cord-blood transplantation in patients with hematologic disorders. We have established a registry containing information on the outcome ofExpand
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Outcome of cord-blood transplantation from related and unrelated donors. Eurocord Transplant Group and the European Blood and Marrow Transplantation Group.
BACKGROUND Cord-blood banks have increased the use of cord-blood transplantation in patients with hematologic disorders. We have established a registry containing information on the outcome ofExpand
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Bone marrow failure in Fanconi anemia is triggered by an exacerbated p53/p21 DNA damage response that impairs hematopoietic stem and progenitor cells.
Fanconi anemia (FA) is an inherited DNA repair deficiency syndrome. FA patients undergo progressive bone marrow failure (BMF) during childhood, which frequently requires allogeneic hematopoietic stemExpand
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