Clinical Biochemistry of Domestic Animals (Sixth Edition) - UMK ...

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Chapter | 24 Lysosomal Storage Diseases
C . Gene Therapy
The most striking clinical results of gene therapy involving
an LSD have been those seen in a series of neonatal gene
transfer studies conducted using viral vectors in the murine
and canine models of MPS VII documenting significant
improvement of cornea, joint, and cardiac disease ( Daly
et al. , 1999, 2001 ; Ponder et al. , 2002 ; Xu et al. , 2002a ,
2002b). In spite of the rarity of MPS VII (1/250,000 live
births in humans), these disease models have become a paradigm
for LSDs in general because of the ability to detect
the normal enzyme (β-glucuronidase) activity directly
using a histochemical technique. Intracranial injection
of vector carrying the feline alpha mannosidase gene has
produced remarkable clinical improvement in affected cats
( Vite et al. , 2005 ).
ACKNOWLEDGMENTS
The authors wish to thank Dr. Donald F. Patterson for his
vision and for establishing the first Section of Medical
Genetics in a veterinary school; Dr. Peter Jezyk for developing
a metabolic screening laboratory for animals; Mr.
James Hayden for the illustrations; Ms. Ping Wang, Ms.
Marisa Van Hoeven, and Ms. Angie Huff for metabolic
and enzyme analyses; Drs. Cathy Just and Meg Weil; Ms.
Patricia O’Donnell; many Medical Genetics residents; and
a cadre of veterinary students for animal care. Supported by
NIH grants RR02512, DK25795, and DK54481.
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