Skip to main content
SpringerLink
Log in

Bone marrow transplantation in young aspartylglucosaminuria mice: improved clearance of lysosomal storage in brain by using wild type as compared to heterozygote donors

  • Correspondence
  • Published:
Bone Marrow Transplantation Submit manuscript

This is a preview of subscription content, log in via an institution to check access.

Access this article

Log in via an institution

Price excludes VAT (USA)
Tax calculation will be finalised during checkout.

Instant access to the full article PDF.

Institutional subscriptions

Figure 1
Figure 2

References

  1. Malatack JJ, Consolini DM, Bayever E . The status of hematopoietic stem cell transplantation in lysosomal storage disease. Pediatr Neurol 2003; 29: 391–403.

    Article  Google Scholar 

  2. Aula P, Jalanko A, Peltonen L . Aspartylglucosaminuria. In: Scriver CR, Beaudet A, Sly W, Valle D, Childs B, Vogelstein B (eds.). The Metabolic and Molecular Basis of Inherited Disease, 8th edn McGrow-Hill: New York, 2001; pp 3535–3550.

    Google Scholar 

  3. Harkke S, Laine M, Jalanko A . Aspartylglucosaminidase (AGA) is efficiently produced and endocytosed by glial cells: implication for the therapy of a lysosomal storage disorder. J Gene Med 2003; 5: 472–482.

    Article  CAS  Google Scholar 

  4. Peltola M, Kyttälä A, Heinonen O et al. Adenovirus-mediated gene transfer results in decreased lysosomal storage in brain and total correction in liver of aspartylglucosaminuria (AGU) mouse. Gene Ther 1998; 5: 1314–1321.

    Article  CAS  Google Scholar 

  5. Jalanko A, Tenhunen K, McKinney CE et al. Mice with an aspartylglucosaminuria mutation similar to humans replicate the pathophysiology in patients. Hum Mol Genet 1998; 7: 265–272.

    Article  CAS  Google Scholar 

  6. Laine M, Richter J, Fahlman C et al. Correction of peripheral lysosomal accumulation in mice with aspartylglucosaminuria by bone marrow transplantation. Exp Hematol 1999; 27: 1467–1474.

    Article  CAS  Google Scholar 

  7. Arvio M, Sauna-Aho O, Peippo M . Bone marrow transplantation for aspartylglucosaminuria: follow-up study of transplanted and non-transplanted patients. J Pediatr 2001; 138: 288–290.

    Article  CAS  Google Scholar 

  8. Tenhunen K, Uusitalo A, Autti T et al. Monitoring the CNS pathology in aspartylglucosaminuria. J Neuropathol Exp Neurol 1998; 57: 1154–1163.

    Article  CAS  Google Scholar 

  9. Bayever E, Ladish S, Philippart M et al. Bone marrow transplantation for metachromatic leukodystrophy. Lancet 1985; 31: 471–473.

    Article  Google Scholar 

  10. Kennedy DW, Abkowitz JL . Kinetics of central nervous system microglial and macrophage engraftment: analysis using a transgenic bone marrow transplantation model. Blood 1997; 90: 986–993.

    CAS  PubMed  Google Scholar 

Download references

Author information

Authors and Affiliations

  1. Department of Molecular Medicine, National Public Health Institute, Biomedicum Helsinki, Helsinki, POB 104, FIN-00251, Finland

    M Laine, L Ahtiainen & A Jalanko

  2. Hospital for Children and Adolescents, University of Helsinki, Finland

    J Rapola

  3. Molecular Medicine and Gene Therapy, Lund University Hospital, Lund, 22184, Sweden

    J Richter

Authors
  1. M Laine
    View author publications

    You can also search for this author in PubMed Google Scholar

  2. L Ahtiainen
    View author publications

    You can also search for this author in PubMed Google Scholar

  3. J Rapola
    View author publications

    You can also search for this author in PubMed Google Scholar

  4. J Richter
    View author publications

    You can also search for this author in PubMed Google Scholar

  5. A Jalanko
    View author publications

    You can also search for this author in PubMed Google Scholar

Rights and permissions

Reprints and permissions

About this article

Cite this article

Laine, M., Ahtiainen, L., Rapola, J. et al. Bone marrow transplantation in young aspartylglucosaminuria mice: improved clearance of lysosomal storage in brain by using wild type as compared to heterozygote donors. Bone Marrow Transplant 34, 1001–1003 (2004). https://doi.org/10.1038/sj.bmt.1704665

Download citation

  • Published:

  • Issue Date:

  • DOI: https://doi.org/10.1038/sj.bmt.1704665

  • Springer Nature Limited

This article is cited by

Search

Navigation