The DOI matches its Crossref record; 30 of the 30 references it cites were found in a registry or an index.
Bone marrow transplantation as effective treatment of central nervous system disease in globoid cell leukodystrophy, metachromatic leukodystrophy, adrenoleukodystrophy, mannosidosis, fucosidosis, aspartylglucosaminuria, Hurler, Maroteaux-Lamy, and Sly syndromes, and Gaucher disease type III
Abstract
Over 400 patients with lysosomal and peroxisomal storage diseases have received hematopoietic stem cell transplantation from normal donors. Without treatment, all of these diseases have an inexorable fate leading to central nervous system deterioration and early death. On the other hand, all of the engrafted hosts have had a remarkable positive clinical improvement in response to normalization of previously deficient enzymatic activity. Survival data for those engrafted indicates continued life-span as long as two decades beyond transplantation. The particular diseases treated in this way are included in this article. The specific indications and methods for transplantation are also included in this article.
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Cites
TAN, P. (2001) · A Clinical Approach to Medicine · doi:10.1142/9789810248178_0024 · Cited by 2,697
Hematopoietic cells differentiate into both microglia and macroglia in the brains of adult mice
Eglitis, M. A., & Mezey, É. (1997) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.94.8.4080 · Cited by 1,016
Azizi, S. A., Stokes, D., Augelli, B. J., DiGirolamo, C., & Prockop, D. J. (1998) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.95.7.3908 · Cited by 909
Neural progenitor cell engraftment corrects lysosomal storage throughout the MRS VII mouse brain
Snyder, E. Y., Taylor, R. M., & Wolfe, J. H. (1995) · Nature · doi:10.1038/374367a0 · Cited by 473
Peters, C., Shapiro, E. G., Anderson, J., Henslee-Downey, P. J., Klemperer, M. R., Cowan, M. J., Saunders, E. F., deAlarcon, P. A., Twist, C., Nachman, J. B., et al. (1998) · Blood · doi:10.1182/blood.v91.7.2601 · Cited by 342
Self-citationPrevention of Lysosomal Storage in Tay-Sachs Mice Treated with N -Butyldeoxynojirimycin
Platt, F. M., Neises, G. R., Reinkensmeier, G., Townsend, M. J., Perry, V. H., Proia, R. L., Winchester, B., Dwek, R. A., & Butters, T. D. (1997) · Science · doi:10.1126/science.276.5311.428 · Cited by 340
Hematopoietic Stem-Cell Transplantation in Globoid-Cell Leukodystrophy
Krivit, W., Shapiro, E. G., Peters, C., Wagner, J. E., Cornu, G., Kurtzberg, J., Wenger, D. A., Kolodny, E. H., Vanier, M. T., Loes, D. J., et al. (1998) · New England Journal of Medicine · doi:10.1056/nejm199804163381605 · Cited by 319
Self-citationOutcome of unrelated donor bone marrow transplantation in 40 children with Hurler syndrome
Peters, C., Balthazor, M., Shapiro, E., King, R., Kollman, C., Hegland, J., Henslee- Downey, J., Trigg, M., Cowan, M., Sanders, J., et al. (1996) · Blood · doi:10.1182/blood.v87.11.4894.bloodjournal87114894 · Cited by 315
Self-citationNeuropsychological outcomes of several storage diseases with and without bone marrow transplantation
Shapiro, E. G., Lockman, L. A., Balthazor, M., & Krivit, W. (1995) · Journal of Inherited Metabolic Disease · doi:10.1007/bf00710053 · Cited by 245
Self-citationAdrenoleukodystrophy: Phenotypic variability and implications for therapy
Moser, H. W., Moser, A. B., Smith, K. D., Bergin, A., Borel, J., Shankroff, J., Stine, O. C., Merette, C., Ott, J., Krivit, W., et al. (1992) · Journal of Inherited Metabolic Disease · doi:10.1007/bf01799621 · Cited by 239
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Horwitz, E. M., Gordon, P. L., Koo, W. K. K., Marx, J. C., Neel, M. D., McNall, R. Y., Muul, L., & Hofmann, T. (2002) · Proceedings of the National Academy of Sciences · doi:10.1073/pnas.132252399 · Cited by 1,650
Cord-Blood Transplants from Unrelated Donors in Patients with Hurler's Syndrome
Staba, S. L., Escolar, M. L., Poe, M., Kim, Y., Martin, P. L., Szabolcs, P., Allison-Thacker, J., Wood, S., Wenger, D. A., Rubinstein, P., et al. (2004) · New England Journal of Medicine · doi:10.1056/nejmoa032613 · Cited by 478
Self-citationCronk, J. C., Filiano, A. J., Louveau, A., Marin, I., Marsh, R., Ji, E., Goldman, D. H., Smirnov, I., Geraci, N., Acton, S., et al. (2018) · Journal of Experimental Medicine · doi:10.1084/jem.20180247 · Cited by 412
Sphingolipid metabolism diseases
Kolter, T., & Sandhoff, K. (2006) · Biochimica et Biophysica Acta (BBA) - Biomembranes · doi:10.1016/j.bbamem.2006.05.027 · Cited by 367
Bone marrow transdifferentiation in brain after transplantation: a retrospective study
Cogle, C. R., Yachnis, A. T., Laywell, E. D., Zander, D. S., Wingard, J. R., Steindler, D. A., & Scott, E. W. (2004) · The Lancet · doi:10.1016/s0140-6736(04)16102-3 · Cited by 305
Metadata
- Authors
- Krivit, W., Peters, C., & Shapiro, E. G.
- Venue
- Current Opinion in Neurology
- Published
- 1999-04
- Volume
- 12
- Issue
- 2
- Pages
- 167-176
- ISSN
- 1350-7540 (ISSN-L)
- OpenAlex
- W2060496255
- Cited by
- 273
- References
- 30
- FWCI
- 6.05
- Type
- journal article
- Language
- en
- Open access
- closed
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- CoChat. (2026). Citation check: Bone marrow transplantation as effective treatment of central nervous system disease in globoid cell leukodystrophy, metachromatic leukodystrophy, adrenoleukodystrophy, mannosidosis, fucosidosis, aspartylglucosaminuria, Hurler, Maroteaux-Lamy, and Sly syndromes, and Gaucher disease type III. https://app.cochat.ai/papers/10.1097/00019052-199904000-00007
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- Krivit, W., Peters, C., & Shapiro, E. G. (1999). Bone marrow transplantation as effective treatment of central nervous system disease in globoid cell leukodystrophy, metachromatic leukodystrophy, adrenoleukodystrophy, mannosidosis, fucosidosis, aspartylglucosaminuria, Hurler, Maroteaux-Lamy, and Sly syndromes, and Gaucher disease type III. Current Opinion in Neurology. https://doi.org/10.1097/00019052-199904000-00007
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Contains information from the Semantic Scholar Academic Graph Datasets, made available under the ODC Attribution License (ODC-BY 1.0).
Semantic Scholar’s data is described in: Kinney, R., et al. (2023). The Semantic Scholar Open Data Platform. arXiv:2301.10140.
