# MARROW TRANSPLANT THERAPY FOR MUCOPOLYSACCHARIDOSIS

> **NIH NIH R01** · COLORADO STATE UNIVERSITY · 1988 · $106,604

## Abstract

We propose to employ a feline model of a lysosomal storage disease,
mucopolysaccharidosis VI [arylsulfatase B (ASB) deficiency], to investigate
prospectively the degree to which various clinicopathologic and biochemical
manifestations of this glycosaminoglycans (GAG) storage disease are
prevented, stabilized or corrected through allogeneic bone marrow
transplantation (BMT).  We have established a breeding colony of
mucopolysaccharidosis VI (MPS VI)-affected (homozygous) and carrier
(heterozygous) cats.  In our initial studies, we have corrected leukocyte
arylsulfatase B deficiency in 3 of 3 cats by BMT.  Following bone marrow
transplantation, many of the clinical features related to excess GAG
storage, e.g. corneal clouding, facial dysmorphia, inability to ambulate,
and excess urinary excretion of GAG have been reversed after donor-origin,
ASB-competent leukocytes have become engrafted.  We propose to extend the
above observations by conducting additional allogeneic bone marrow
transplants in MPS VI affected cats to determine the mechanism by which BMT
may prevent, stabilize, or reverse manifestations of MPS VI and the
importance of age of MPS-affected individual at time of BMT in influencing
the efficacy of this treatment.  We will perform BMT in 3 groups of 5 MPS
VI-affected cats each at the ages of 12 weeks, 6 months, and 1 year and
monitor the following parameters pre- and post-BMT in cats:  Radiography
and echocardiography and biomicroscopy to evaluate effects of GAG
accumulation in skeletal cardiac, corneal, and other visceral tissues; and
light and electron microscopy accumulation to localize the intracellular
and interstitial of GAG in tissues and lesions; leukocyte, tissue, and
urinary arylsulfatase B activity will be assessed to determine whether
engraftment has occurred and the degree to which enzyme-competent cells
will reverse the GAC accumulation and lesions in affected target tissues.
Five MPS VI-affected not treated by BMT cats will be used as controls.  We
also propose to determine the effect of MPS VI affected allogeneic bone
marrow transplantation into unaffected normal recipients to ascertain the
extent to which bone marrow derived cells are responsible for
glycosaminoglycans degradation in various tissues and organs.  Results from
these studies will provide information directly pertinent to therapy of
lysosomal storage disorders of humans by bone marrow transplantation.

## Key facts

- **NIH application ID:** 3157942
- **Project number:** 5R01AR037095-03
- **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:** 1988
- **Award amount:** $106,604
- **Award type:** 5
- **Project period:** 1986-04-01 → 1989-03-31

## Primary source

NIH RePORTER: https://reporter.nih.gov/project-details/3157942

## Citation

> US National Institutes of Health, RePORTER application 3157942, MARROW TRANSPLANT THERAPY FOR MUCOPOLYSACCHARIDOSIS (5R01AR037095-03). Retrieved via MORVS 2026-10-24 from https://api.morvs.ai/grant/nih/3157942. Licensed CC0.

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