ArticleBlood2015
Neonatal bone marrow transplantation prevents bone pathology in a mouse model of mucopolysaccharidosis type I.
Article in Blood, 2015. The graph could read no effect estimate from its abstract, so it casts no vote on the map. Cited by 31 papers.
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The abstract states no effect estimate the extractor could read, or names no intervention and outcome on the map, so this paper lights no cell and moves no belief. It is still indexed, cited and linked below.
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Who cites it
31 citing papers in PubMed, 43 citations in OpenAlex.
- Neonatal gene therapy effectively prevents disease manifestations in a murine model of Mucopolysaccharidosis type I.Molecular therapy. Methods & clinical development · 2025Article
- Article
- Antibodies to recombinant human alpha-L-iduronidase prevent disease correction in cortical bone in MPS I mice.Molecular therapy. Methods & clinical development · 2025Article
- Lentiviral Vector-MediatedHuman gene therapy · 2024Article
- Body Height of MPS I and II Patients after Hematopoietic Stem Cell Transplantation: The Impact of Dermatan Sulphate.Diagnostics (Basel, Switzerland) · 2024Article
- MPSI Manifestations and Treatment Outcome: Skeletal Focus.International journal of molecular sciences · 2022Review
- In utero adenine base editing corrects multi-organ pathology in a lethal lysosomal storage disease.Nature communications · 2021Article
- Dysostosis in mucopolysaccharidosis type 2: A case of longitudinal follow up and literature review.Radiology case reports · 2021Article
- Mucopolysaccharidosis Type I: Current Treatments, Limitations, and Prospects for Improvement.Biomolecules · 2021Review
- Article
- Failures of Endochondral Ossification in the Mucopolysaccharidoses.Current osteoporosis reports · 2020Review
- Review
- Intravenous Enzyme Replacement Therapy in Mucopolysaccharidoses: Clinical Effectiveness and Limitations.International journal of molecular sciences · 2020Review
- Article
- Pre-clinical Mouse Models of Neurodegenerative Lysosomal Storage Diseases.Frontiers in molecular biosciences · 2020Review
- Hematopoietic Stem Cell Transplantation for Mucopolysaccharidoses: Past, Present, and Future.Biology of blood and marrow transplantation : journal of the American Society for Blood and Marrow Transplantation · 2019Review
- Biomarkers in patients with mucopolysaccharidosis type II and IV.Molecular genetics and metabolism reports · 2019Article
- Lipid-induced lysosomal damage after demyelination corrupts microglia protective function in lysosomal storage disorders.The EMBO journal · 2019Article
- Long-term outcomes of systemic therapies for Hurler syndrome: an international multicenter comparison.Genetics in medicine : official journal of the American College of Medical Genetics · 2018Article
- Circulating monocytes accelerate acute liver failure by IL-6 secretion in monkey.Journal of cellular and molecular medicine · 2018Article
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Authors and funding
12 authors at 4 institutions in 2 countries.
Funding
Abstract
Neonatal bone marrow transplantation (BMT) could offer a novel therapeutic opportunity for genetic disorders by providing sustainable levels of the missing protein at birth, thus preventing tissue damage. We tested this concept in mucopolysaccharidosis type I (MPS IH; Hurler syndrome), a lysosomal storage disorder caused by deficiency of α-l-iduronidase. MPS IH is characterized by a broad spectrum of clinical manifestations, including severe progressive skeletal abnormalities. Although BMT increases the life span of patients with MPS IH, musculoskeletal manifestations are only minimally responsive if the timing of BMT delays, suggesting already irreversible bone damage. In this study, we tested the hypothesis that transplanting normal BM into newborn MPS I mice soon after birth can prevent skeletal dysplasia. We observed that neonatal BMT was effective at restoring α-l-iduronidase activity and clearing elevated glycosaminoglycans in blood and multiple organs. At 37 weeks of age, we observed an almost complete normalization of all bone tissue parameters, using radiographic, microcomputed tomography, biochemical, and histological analyses. Overall, the magnitude of improvements correlated with the extent of hematopoietic engraftment. We conclude that BMT at a very early stage in life markedly reduces signs and symptoms of MPS I before they appear.
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Registered trials
Read under generation 80e0d062 · epoch 390. Bibliography from PubMed, PubMed Central and OpenAlex; grants from NIH RePORTER; trial links from ClinicalTrials.gov; estimates, votes and beliefs from the OpenQuestion graph.