Osteogenesis Imperfecta

Diagnosis

The diagnosis is easy when a patient has blue sclerae and a parent with similar eyes. But sometimes the bone disease in the parent can be mild, and sometimes the mutation started with an individual patient. In these cases the collagen production from skin can be measured. Soon it will be possible to check an individual's DNA to see if there is a mutation in the collagen gene.

One kind of skin cell (the fibroblast) also makes type I collagen. A small skin biopsy can be cultured, and the collagen made by the fibroblast is characterized. Patients with osteogenesis imperfecta may have abnormal collagen, or they make make smaller amounts of collagen than normal persons.


Image of gel from collagen studies awaiting permission

Collagen synthesis

This animation reviews the collagen synthesis in normals, which was described in the matrix section,
and compares it to the abnormal collagen synthesis in patients with osteogenesis imperfecta.


The following sections are not yet finished! This is the planned outline:

Histology

Transgenic mice

Osteogenesis imperfecta was one of the first hereditary disease that was artifically created in mice by the technique of replacing a gene inside the cells of an embryo.

Treatment

Bisphosphonates, estrogen, surgery . . .not good enough!

Gene therapy: break-through research

. . . with hope for the future.

Some references

Osteogenesis Imperfecta Foundation

Chamberlain, J. R.(2004). Gene targeting in stem cells from individuals with osteogenesis imperfecta. Science 303: 1198-201.

Pace, J. M.(2001). Deletions and duplications of Gly-Xaa-Yaa triplet repeats in the triple helical domains of type I collagen chains disrupt helix formation and result in several types of osteogenesis imperfecta. Hum Mutat 18: 319-26.

Byers, P. H.(2000). Osteogenesis imperfecta: perspectives and opportunities. Curr Opin Pediatr 12: 603-9.

Prockop, D. J.(2004). Targeting gene therapy for osteogenesis imperfecta. N Engl J Med 350: 2302-4.

Bonadio, J.(1990). Transgenic mouse model of the mild dominant form of osteogenesis imperfecta. Proc Natl Acad Sci U S A 87: 7145-9.


©2004 by Susan Ott
Last update 9/29/04

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