# MARROW TRANSPLANT THERAPY FOR MUCOPOLYSACCHARIDOSIS

> **NIH NIH R01** · COLORADO STATE UNIVERSITY · 1990 · $155,505

## Abstract

Bone marrow transplantation is a promising therapy for some lysosomal
storage disorders in human beings.  Donor leukocytes may establish enzyme
activity on a continual basis in amounts sufficient to be within normal
limits.  Allogeneic bone marrow transplants in animals with lysosomal
storage diseases will determine the degree by which BMT may prevent,
stabilize, or reverse manifestations of various lysosomal storage diseases.
We propose to continue to employ a feline model of mucopolysaccharidosis VI
(arylsulfatase B deficiency) to investigate prospectively the degree to
which various clinical, morphological, and biochemical manifestations of
this glycosaminoglycan storage disease are prevented, stabilized, or
corrected through allogenic bone marrow transplantation.  Long term
evaluation of treated animal is necessary to determine if substrate is
reduced in liver, cartilage, cornea, and other tissues.  Clinical
heterogeneity is marked in animals and people with many of the lysosomal
storage diseases, making clinical evaluation of the progression of disease
subjective.

It is proposed to study larger numbers of BMT treated and untreated affected
animals to determine if differences are statistically significant.  Six MPS
VI affected-BMT treated cats, two MPS VI affected-BMT treated cats which
autologously recovered their own marrow, and 8 MPS VI affected non BMT
treated cats will continue to be monitored.  In addition, six 10 weeks old
MPS VI affected cats will be transplanted in year 4, and six in year 5, for
a total of 18 MPS VI affected-BMT treated cats.  Two additional MPS VI
affected-non treated controls will be monitored beginning year 4.  MPS VI
affected cats will be monitored morphologically pre-and at 6 months then
yearly post-BMT by radiography, photography, computed tomography, slit lamp
biomicroscopy, electroretinography, and light and electron microscopy of
liver, brain, cartilage, and skin biopsies and necropsy tissues.  MPS VI
cats will be monitored biochemically by leukocyte and tissue arylsulfatase B
activity and by analysis and characterization of urine, liver, cornea,
cartilage and cochlea glycosaminoglycans.  Untreated cats will be monitored
similarly for comparison.  These studies will determine the degree to which
enzyme-competent cells will reverse or prevent the glycosaminoglycan
accumulation and lesions in affected target tissues.  Results from these
studies will provide information directly pertinent to therapy of lysosomal
storage disorders of humans by either bone marrow transplantation or gene
therapy.

## Key facts

- **NIH application ID:** 3157937
- **Project number:** 2R01AR037095-04A1
- **Recipient organization:** COLORADO STATE UNIVERSITY
- **SAM.gov UEI:** LT9CXX8L19G1
- **Principal Investigator:** MARY A THRALL
- **Activity code:** R01 (R01, R21, SBIR, etc.)
- **Funding institute:** NIH
- **Fiscal year:** 1990
- **Award amount:** $155,505
- **Award type:** 2
- **Project period:** 1986-04-01 → 1992-11-30

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/3157937

## Citation

> US National Institutes of Health, RePORTER application 3157937, MARROW TRANSPLANT THERAPY FOR MUCOPOLYSACCHARIDOSIS (2R01AR037095-04A1). Retrieved via MORVS 2026-10-25 from https://api.morvs.ai/grant/nih/3157937. Licensed CC0.

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