Jove
Visualize
Contact Us
JoVE
x logofacebook logolinkedin logoyoutube logo
ABOUT JoVE
OverviewLeadershipBlogJoVE Help Center
AUTHORS
Publishing ProcessEditorial BoardScope & PoliciesPeer ReviewFAQSubmit
LIBRARIANS
TestimonialsSubscriptionsAccessResourcesLibrary Advisory BoardFAQ
RESEARCH
JoVE JournalMethods CollectionsJoVE Encyclopedia of ExperimentsArchive
EDUCATION
JoVE CoreJoVE BusinessJoVE Science EducationJoVE Lab ManualFaculty Resource CenterFaculty Site
Terms & Conditions of Use
Privacy Policy
Policies

Related Experiment Videos

Bone-marrow transplantation for metachromatic leucodystrophy.

E Bayever, S Ladisch, M Philippart

    Lancet (London, England)
    |August 31, 1985
    PubMed
    Summary

    Bone marrow transplant (BMT) showed promise for treating a child with metachromatic leukodystrophy. Donor cells in cerebrospinal fluid and continued development suggest BMT

    Related Concept Videos

    You might also read

    Related Articles

    Articles linked to this work by shared authors, journal, and citation graph.

    Sort by
    Same author

    Chronic hepatitis E in immunosuppressed patients: a comprehensive review of the literature.

    Acta gastro-enterologica Belgica·2026
    Same author

    Immune checkpoint inhibitor refractory colitis leading to total colectomy in a melanoma patient.

    Acta gastro-enterologica Belgica·2023
    Same author

    Oral Delivery of Therapeutic Proteins and Peptides: An Overview of Current Technologies and Recommendations for Bridging from Approved Intravenous or Subcutaneous Administration to Novel Oral Regimens.

    Drug research·2015
    Same author

    Continuous infusion of antisense phosphorothioate therapeutics.

    Methods in molecular medicine·2011
    Same author

    Genotype-phenotype correlation in vanishing white matter disease.

    Neurology·2010
    Same author

    Nerve cuff stimulation and the effect of fascicular organization for hand grasp in nonhuman primates.

    Annual International Conference of the IEEE Engineering in Medicine and Biology Society. IEEE Engineering in Medicine and Biology Society. Annual International Conference·2009

    Area of Science:

    • Neurology
    • Hematology
    • Genetics

    Background:

    • Late infantile metachromatic leukodystrophy is a rare genetic disorder affecting the central nervous system.
    • Bone marrow transplantation (BMT) is a potential therapeutic option for certain inherited metabolic diseases.

    Observation:

    • An 11-month-old boy diagnosed with metachromatic leukodystrophy received a bone marrow transplant from an HLA-identical sibling.
    • Cerebrospinal fluid analysis 6 months post-transplant revealed exclusively donor-derived leukocytes.

    Findings:

    • The patient demonstrated sustained developmental progress 33 months after the bone marrow transplant.
    • Successful engraftment of donor cells in the central nervous system was confirmed.

    Implications:

    Related Experiment Videos

    • Bone marrow transplantation may be an effective treatment strategy for specific congenital metabolic disorders impacting the central nervous system.
    • This case highlights the potential of BMT in managing neurodegenerative genetic conditions.