Published in N Engl J Med on May 06, 2004
Cord Blood Stem Cell Transplantation Study (COBLT) | NCT00000603
Cord Blood for Neonatal Hypoxic-ischemic Encephalopathy | NCT00593242
Sanfilippo syndrome: a mini-review. J Inherit Metab Dis (2008) 2.35
Outcomes of transplantation using various hematopoietic cell sources in children with Hurler syndrome after myeloablative conditioning. Blood (2013) 1.91
Intravenous administration of auto serum-expanded autologous mesenchymal stem cells in stroke. Brain (2011) 1.73
Intrathecal enzyme replacement therapy: successful treatment of brain disease via the cerebrospinal fluid. Mol Genet Metab (2007) 1.71
New therapeutic options for lysosomal storage disorders: enzyme replacement, small molecules and gene therapy. Hum Genet (2006) 1.65
Neonatal bone marrow transplantation prevents bone pathology in a mouse model of mucopolysaccharidosis type I. Blood (2014) 1.46
Whole body correction of mucopolysaccharidosis IIIA by intracerebrospinal fluid gene therapy. J Clin Invest (2013) 1.44
Untying the Gordian knot: policies, practices, and ethical issues related to banking of umbilical cord blood. J Clin Invest (2005) 1.43
[Gaucher disease, Fabry disease and mucopolysaccharidosis type I--how can the rheumatologist recognise these patients?]. Z Rheumatol (2006) 1.42
Feasibility of autologous cord blood cells for infants with hypoxic-ischemic encephalopathy. J Pediatr (2013) 1.41
Mesenchymal stem cells derived from peripheral blood protects against ischemia. J Neurotrauma (2007) 1.33
Gene therapy augments the efficacy of hematopoietic cell transplantation and fully corrects mucopolysaccharidosis type I phenotype in the mouse model. Blood (2010) 1.33
Lysosomal storage disorders in the newborn. Pediatrics (2009) 1.29
Gene therapy for mucopolysaccharidosis. Expert Opin Biol Ther (2007) 1.28
Allogeneic stem cell transplantation for the treatment of lysosomal and peroxisomal metabolic diseases. Springer Semin Immunopathol (2004) 1.27
The role of enzyme replacement therapy in severe Hunter syndrome-an expert panel consensus. Eur J Pediatr (2011) 1.27
Advances in the treatment of mucopolysaccharidosis type I. N Engl J Med (2004) 1.25
Long-term follow-up after gene therapy for canavan disease. Sci Transl Med (2012) 1.22
Safety and feasibility of countering neurological impairment by intravenous administration of autologous cord blood in cerebral palsy. J Transl Med (2012) 1.16
Hematopoietic differentiation of induced pluripotent stem cells from patients with mucopolysaccharidosis type I (Hurler syndrome). Blood (2010) 1.15
Radiographic evaluation of bones and joints in mucopolysaccharidosis I and VII dogs after neonatal gene therapy. Mol Genet Metab (2008) 1.15
Adult stem cells and their trans-differentiation potential--perspectives and therapeutic applications. J Mol Med (Berl) (2008) 1.12
Brain conditioning is instrumental for successful microglia reconstitution following hematopoietic stem cell transplantation. Proc Natl Acad Sci U S A (2012) 1.12
Update on umbilical cord blood transplantation. Curr Opin Pediatr (2009) 1.08
Optimization of a therapeutic protocol for intravenous injection of human mesenchymal stem cells after cerebral ischemia in adult rats. Brain Res (2008) 1.08
Mucopolysaccharidosis I, II, and VI: Brief review and guidelines for treatment. Genet Mol Biol (2010) 1.07
Trends in haematopoietic cell transplantation for inborn errors of metabolism. J Inherit Metab Dis (2006) 1.06
Progression of multiple behavioral deficits with various ages of onset in a murine model of Hurler syndrome. Brain Res (2007) 1.05
The Cord Blood Apgar: a novel scoring system to optimize selection of banked cord blood grafts for transplantation (CME). Transfusion (2011) 1.05
Neurodegeneration and cell replacement. Philos Trans R Soc Lond B Biol Sci (2008) 1.01
Reprogramming erythroid cells for lysosomal enzyme production leads to visceral and CNS cross-correction in mice with Hurler syndrome. Proc Natl Acad Sci U S A (2009) 1.00
Mankind's first natural stem cell transplant. J Cell Mol Med (2010) 0.99
Improved retroviral vector design results in sustained expression after adult gene therapy in mucopolysaccharidosis I mice. J Gene Med (2008) 0.99
Orthopaedic management of Hurler's disease after hematopoietic stem cell transplantation: a systematic review. J Inherit Metab Dis (2011) 0.98
Cord blood in regenerative medicine: do we need immune suppression? J Transl Med (2007) 0.97
Paracrine molecules of mesenchymal stem cells for hematopoietic stem cell niche. Bone Marrow Res (2011) 0.96
Glycosaminoglycan storage disorders: a review. Biochem Res Int (2011) 0.96
Four successful pregnancies in a patient with mucopolysaccharidosis type I treated by allogeneic bone marrow transplantation. J Inherit Metab Dis (2009) 0.95
Concise review: umbilical cord blood transplantation: past, present, and future. Stem Cells Transl Med (2014) 0.95
Bone marrow transplantation for feline mucopolysaccharidosis I. Mol Genet Metab (2007) 0.95
Engineering a lysosomal enzyme with a derivative of receptor-binding domain of apoE enables delivery across the blood-brain barrier. Proc Natl Acad Sci U S A (2013) 0.94
Research challenges in central nervous system manifestations of inborn errors of metabolism. Mol Genet Metab (2010) 0.93
Supporting family adaptation to presymptomatic and "untreatable" conditions in an era of expanded newborn screening. J Pediatr Psychol (2008) 0.92
Umbilical cord blood donation: public or private? Bone Marrow Transplant (2015) 0.91
Clinical outcomes following hematopoietic stem cell transplantation for the treatment of mucopolysaccharidosis VI. Mol Genet Metab (2010) 0.91
Methods of neurodevelopmental assessment in children with neurodegenerative disease: Sanfilippo syndrome. JIMD Rep (2013) 0.90
Mucopolysaccharidosis I: Alpha-L-Iduronidase mutations in three Tunisian families. J Inherit Metab Dis (2005) 0.90
An exploratory study of brain function and structure in mucopolysaccharidosis type I: long term observations following hematopoietic cell transplantation (HCT). Mol Genet Metab (2012) 0.90
Impact of enzyme replacement therapy and hematopoietic stem cell transplantation in patients with Morquio A syndrome. Drug Des Devel Ther (2015) 0.89
Therapies for the bone in mucopolysaccharidoses. Mol Genet Metab (2014) 0.89
Long-term therapeutic efficacy of allogenic bone marrow transplantation in a patient with mucopolysaccharidosis IVA. Mol Genet Metab Rep (2015) 0.88
Short-term growth hormone treatment in children with Hurler syndrome after hematopoietic cell transplantation. Bone Marrow Transplant (2009) 0.87
Current and emerging management options for patients with Morquio A syndrome. Ther Clin Risk Manag (2013) 0.87
alpha-L-iduronidase therapy for mucopolysaccharidosis type I. Biologics (2008) 0.87
The craniocervical junction following successful haematopoietic stem cell transplantation for mucopolysaccharidosis type I H (Hurler syndrome). J Inherit Metab Dis (2011) 0.85
The role of microglia in human disease: therapeutic tool or target? Acta Neuropathol (2014) 0.85
A Prospective Natural History Study of Mucopolysaccharidosis Type IIIA. J Pediatr (2016) 0.85
Newborn screening for neuropathic lysosomal storage disorders. J Inherit Metab Dis (2010) 0.85
Innovative therapy for Classic Galactosemia - tale of two HTS. Mol Genet Metab (2011) 0.85
Use of a precious resource: parental decision making about using autologous umbilical cord blood in studies involving young children with type 1 diabetes. Contemp Clin Trials (2011) 0.84
Treatment of hip dysplasia in patients with mucopolysaccharidosis type I after hematopoietic stem cell transplantation: results of an international consensus procedure. Orphanet J Rare Dis (2013) 0.83
Growth patterns and the use of growth hormone in the mucopolysaccharidoses. J Pediatr Rehabil Med (2010) 0.83
Growth, final height and endocrine sequelae in a UK population of patients with Hurler syndrome (MPS1H). J Inherit Metab Dis (2011) 0.82
Long-term functional outcomes of children with hurler syndrome treated with unrelated umbilical cord blood transplantation. JIMD Rep (2015) 0.82
Cell- and gene-based therapeutic approaches for neurological deficits in mucopolysaccharidoses. Curr Pharm Biotechnol (2011) 0.82
Hematopoietic stem cell transplantation for Morquio A syndrome. Mol Genet Metab (2015) 0.82
The Neurobehavioral Phenotype in Mucopolysaccharidosis Type IIIB: an Exploratory Study. Mol Genet Metab Rep (2016) 0.82
Clinical manifestations and treatment of mucopolysaccharidosis type I patients in Latin America as compared with the rest of the world. J Inherit Metab Dis (2011) 0.82
Increased longevity and metabolic correction following syngeneic BMT in a murine model of mucopolysaccharidosis type I. Bone Marrow Transplant (2011) 0.82
Clinical aspects of neuropathic lysosomal storage disorders. J Inherit Metab Dis (2010) 0.81
Rheumatologic aspects of lysosomal storage diseases. Clin Rheumatol (2006) 0.80
Co-expression of MGMT(P140K) and alpha-L-iduronidase in primary hepatocytes from mucopolysaccharidosis type I mice enables efficient selection with metabolic correction. J Gene Med (2008) 0.80
Gene therapy for neurologic manifestations of mucopolysaccharidoses. Expert Opin Drug Deliv (2014) 0.79
Molecular Genetics and Metabolism Report Long-term follow-up of post hematopoietic stem cell transplantation for Hurler syndrome: clinical, biochemical, and pathological improvements. Mol Genet Metab Rep (2015) 0.79
Mucopolysaccharidosis (MPS) Physical Symptom Score: Development, Reliability, and Validity. JIMD Rep (2015) 0.78
Retroviral-vector-mediated gene therapy to mucopolysaccharidosis I mice improves sensorimotor impairments and other behavioral deficits. J Inherit Metab Dis (2012) 0.78
Neonatal cellular and gene therapies for mucopolysaccharidoses: the earlier the better? J Inherit Metab Dis (2015) 0.78
Comparison of Endovascular and Intraventricular Gene Therapy With Adeno-Associated Virus-α-L-Iduronidase for Hurler Disease. Neurosurgery (2014) 0.77
The Role of HLA in Cord Blood Transplantation. Bone Marrow Res (2012) 0.77
Cardiac disease in mucopolysaccharidosis type I attributed to catecholaminergic and hemodynamic deficiencies. Am J Physiol Heart Circ Physiol (2010) 0.77
Normalization and improvement of CNS deficits in mice with Hurler syndrome after long-term peripheral delivery of BBB-targeted iduronidase. Mol Ther (2014) 0.77
Cord-blood transplants from unrelated donors in Hurler's syndrome. N Engl J Med (2004) 0.76
TCRαβ CD19 depletion in allogeneic haematopoietic stem cell transplantation performed for Hurler syndrome. Bone Marrow Transplant (2015) 0.76
Ethical and policy issues relating to progenitor-cell-based strategies for prevention of atherosclerosis. J Med Ethics (2007) 0.75
Twenty years of progress in biogerontology research. Age (Dordr) (2006) 0.75
Precocious initiation of spermatogenesis in a 19-month-old boy with Hurler syndrome. Basic Clin Androl (2014) 0.75
Neonatal tolerance induction enables accurate evaluation of gene therapy for MPS I in a canine model. Mol Genet Metab (2016) 0.75
Enzyme replacement therapy in Hurler syndrome after failure of hematopoietic transplant. Mol Genet Metab Rep (2015) 0.75
Implication of cord blood for cell-based therapy in refractory childhood diseases. Int J Stem Cells (2010) 0.75
Mortality after hematopoietic stem cell transplantation for severe mucopolysaccharidosis type I: the 30-year University of Minnesota experience. J Inherit Metab Dis (2017) 0.75
The effects of early and late bone marrow transplantation in siblings with alpha-mannosidosis. Is early haematopoietic cell transplantation the preferred treatment option? J Inherit Metab Dis (2010) 0.75
Outcomes after transplantation of cord blood or bone marrow from unrelated donors in adults with leukemia. N Engl J Med (2004) 7.10
Outcomes of transplantation of unrelated donor umbilical cord blood and bone marrow in children with acute leukaemia: a comparison study. Lancet (2007) 6.93
Infusion of ex vivo expanded T regulatory cells in adults transplanted with umbilical cord blood: safety profile and detection kinetics. Blood (2010) 5.79
Control of T(H)17/T(reg) balance by hypoxia-inducible factor 1. Cell (2011) 5.63
Immortalization of human bronchial epithelial cells in the absence of viral oncoproteins. Cancer Res (2004) 5.57
Ancestry and pharmacogenomics of relapse in acute lymphoblastic leukemia. Nat Genet (2011) 4.93
Transplantation of umbilical-cord blood in babies with infantile Krabbe's disease. N Engl J Med (2005) 4.26
Magnetic resonance imaging and head circumference study of brain size in autism: birth through age 2 years. Arch Gen Psychiatry (2005) 3.94
Lineage-specific gene duplication and loss in human and great ape evolution. PLoS Biol (2004) 3.45
One-unit versus two-unit cord-blood transplantation for hematologic cancers. N Engl J Med (2014) 3.07
Newborn screening for Krabbe disease: the New York State model. Pediatr Neurol (2009) 2.88
A method for calling gains and losses in array CGH data. Biostatistics (2005) 2.86
Optimizing donor selection for public cord blood banking: influence of maternal, infant, and collection characteristics on cord blood unit quality. Transfusion (2013) 2.19
Partial inhibition of integrin alpha(v)beta6 prevents pulmonary fibrosis without exacerbating inflammation. Am J Respir Crit Care Med (2007) 2.17
Krabbe disease treated with hematopoietic stem cell transplantation: serial assessment of anisotropy measurements--initial experience. Radiology (2005) 2.10
The long-term outcomes of presymptomatic infants transplanted for Krabbe disease: report of the workshop held on July 11 and 12, 2008, Holiday Valley, New York. Genet Med (2009) 2.07
Long-term follow-up of endurance and safety outcomes during enzyme replacement therapy for mucopolysaccharidosis VI: Final results of three clinical studies of recombinant human N-acetylgalactosamine 4-sulfatase. Mol Genet Metab (2008) 2.04
Pramipexole vs levodopa as initial treatment for Parkinson disease: a 4-year randomized controlled trial. Arch Neurol (2004) 2.02
Dust mite, cockroach, cat, and dog allergen concentrations in homes of asthmatic children in the northeastern United States: impact of socioeconomic factors and population density. Environ Health Perspect (2002) 1.99
Antimetabolite therapy for lesser-risk B-lineage acute lymphoblastic leukemia of childhood: a report from Children's Oncology Group Study P9201. Blood (2007) 1.97
Randomized, double-blind trial of fluconazole versus voriconazole for prevention of invasive fungal infection after allogeneic hematopoietic cell transplantation. Blood (2010) 1.97
On the nature of cardiovascular activation at an arousal from sleep. Sleep (2003) 1.96
Multicenter study of banked third-party virus-specific T cells to treat severe viral infections after hematopoietic stem cell transplantation. Blood (2013) 1.95
STAT3 regulates arginase-I in myeloid-derived suppressor cells from cancer patients. J Clin Invest (2013) 1.94
Enzyme replacement therapy in mucopolysaccharidosis VI (Maroteaux-Lamy syndrome). J Pediatr (2004) 1.94
Augmentation of umbilical cord blood (UCB) transplantation with ex vivo-expanded UCB cells: results of a phase 1 trial using the AastromReplicell System. Blood (2003) 1.92
Outcomes of transplantation using various hematopoietic cell sources in children with Hurler syndrome after myeloablative conditioning. Blood (2013) 1.91
Genome-wide association study identifies germline polymorphisms associated with relapse of childhood acute lymphoblastic leukemia. Blood (2012) 1.90
Hepatic veno-occlusive disease following stem cell transplantation: incidence, clinical course, and outcome. Biol Blood Marrow Transplant (2009) 1.87
An introductory characterization of a combat-casualty-care relevant swine model of closed head injury resulting from exposure to explosive blast. J Neurotrauma (2009) 1.86
Endpoints for clinical trials testing treatment of acute graft-versus-host disease: a joint statement. Biol Blood Marrow Transplant (2009) 1.84
Related umbilical cord blood transplantation in patients with thalassemia and sickle cell disease. Blood (2002) 1.83
Novel susceptibility variants at 10p12.31-12.2 for childhood acute lymphoblastic leukemia in ethnically diverse populations. J Natl Cancer Inst (2013) 1.81
Cerebral X-linked adrenoleukodystrophy: the international hematopoietic cell transplantation experience from 1982 to 1999. Blood (2004) 1.79
Unrelated cord blood transplantation in children with sickle cell disease: review of four-center experience. Pediatr Transplant (2007) 1.76
Phase II study of nelarabine (compound 506U78) in children and young adults with refractory T-cell malignancies: a report from the Children's Oncology Group. J Clin Oncol (2005) 1.76
Effect of HLA-matching recipients to donor noninherited maternal antigens on outcomes after mismatched umbilical cord blood transplantation for hematologic malignancy. Biol Blood Marrow Transplant (2012) 1.72
Differences in quality between privately and publicly banked umbilical cord blood units: a pilot study of autologous cord blood infusion in children with acquired neurologic disorders. Transfusion (2010) 1.70
Successful immune reconstitution decreases leukemic relapse and improves survival in recipients of unrelated cord blood transplantation. Biol Blood Marrow Transplant (2006) 1.65
In vivo imaging of tumor response to therapy using a dual-modality imaging strategy. Int J Cancer (2006) 1.62
Ectopic expression of the proto-oncogene Mer in pediatric T-cell acute lymphoblastic leukemia. Clin Cancer Res (2006) 1.61
Methylation variation at IGF2 differentially methylated regions and maternal folic acid use before and during pregnancy. Epigenetics (2011) 1.60
Limitations of dual time point PET in the assessment of lung nodules with low FDG avidity. Lung Cancer (2009) 1.59
Embedding concepts of sex and gender health differences into medical curricula. J Womens Health (Larchmt) (2013) 1.59
Replacing acid alpha-glucosidase in Pompe disease: recombinant and transgenic enzymes are equipotent, but neither completely clears glycogen from type II muscle fibers. Mol Ther (2005) 1.59
Threshold effect of urinary glycosaminoglycans and the walk test as indicators of disease progression in a survey of subjects with Mucopolysaccharidosis VI (Maroteaux-Lamy syndrome). Am J Med Genet A (2005) 1.58
Efficacy and safety of ex vivo cultured adult human mesenchymal stem cells (Prochymal™) in pediatric patients with severe refractory acute graft-versus-host disease in a compassionate use study. Biol Blood Marrow Transplant (2010) 1.56
Allergic reactions to E. coli L-asparaginase do not affect outcome in childhood B-precursor acute lymphoblastic leukemia: a Children's Oncology Group Study. J Pediatr Hematol Oncol (2007) 1.56
Association between rectal optical signatures and colonic neoplasia: potential applications for screening. Cancer Res (2009) 1.53
A perspective on the selection of unrelated donors and cord blood units for transplantation. Blood (2012) 1.53
Computed tomography predictors of response to endobronchial valve lung reduction treatment. Comparison with Chartis. Am J Respir Crit Care Med (2015) 1.48
Umbilical cord blood: a guide for primary care physicians. Am Fam Physician (2011) 1.47
Total colony-forming units are a strong, independent predictor of neutrophil and platelet engraftment after unrelated umbilical cord blood transplantation: a single-center analysis of 435 cord blood transplants. Biol Blood Marrow Transplant (2011) 1.46
Results of the cord blood transplantation study (COBLT): outcomes of unrelated donor umbilical cord blood transplantation in pediatric patients with lysosomal and peroxisomal storage diseases. Biol Blood Marrow Transplant (2006) 1.45
Improved outcomes in liver transplantation in children with acute liver failure. J Pediatr Gastroenterol Nutr (2014) 1.45
Inefficient dystrophin expression after cord blood transplantation in Duchenne muscular dystrophy. Muscle Nerve (2010) 1.45
Paternal obesity is associated with IGF2 hypomethylation in newborns: results from a Newborn Epigenetics Study (NEST) cohort. BMC Med (2013) 1.44
Metal oxide thin film phototransistor for remote touch interactive displays. Adv Mater (2012) 1.43
ARID5B genetic polymorphisms contribute to racial disparities in the incidence and treatment outcome of childhood acute lymphoblastic leukemia. J Clin Oncol (2012) 1.39
Complete DiGeorge syndrome: development of rash, lymphadenopathy, and oligoclonal T cells in 5 cases. J Allergy Clin Immunol (2004) 1.39
Hematopoietic recovery after unrelated umbilical cord-blood allogeneic transplantation in adults treated with in vivo stem cell factor (R-MetHuSCF) and filgrastim administration. Leuk Res (2003) 1.39
DTI registration in atlas based fiber analysis of infantile Krabbe disease. Neuroimage (2011) 1.38
In vitro priming and expansion of cytomegalovirus-specific Th1 and Tc1 T cells from naive cord blood lymphocytes. Blood (2006) 1.37
Unrelated umbilical cord blood transplantation in adult patients. Biol Blood Marrow Transplant (2003) 1.37
Urokinase receptors are required for alpha 5 beta 1 integrin-mediated signaling in tumor cells. J Biol Chem (2006) 1.37
Alternative pathway of complement in children with diarrhea-associated hemolytic uremic syndrome. Clin J Am Soc Nephrol (2009) 1.36
Umbilical cord blood collection: do patients really understand? J Perinat Med (2007) 1.35
Defibrotide for the treatment of severe hepatic veno-occlusive disease and multiorgan failure after stem cell transplantation: a multicenter, randomized, dose-finding trial. Biol Blood Marrow Transplant (2010) 1.34
New adoptive immunotherapy strategies for solid tumours with CIK cells. Expert Opin Biol Ther (2012) 1.33
Mutation analysis of feline Niemann-Pick C1 disease. Mol Genet Metab (2003) 1.33
Risk factor analysis of outcomes after unrelated cord blood transplantation in patients with hurler syndrome. Biol Blood Marrow Transplant (2009) 1.32
The effects of normal aging on sleep spindle and K-complex production. Clin Neurophysiol (2002) 1.31
Marrow cell transplantation for infantile hypophosphatasia. J Bone Miner Res (2003) 1.31