Human Therapy

The researchers' work suggests that neural progenitor cells could eventually be used to treat Sly Disease in humans.

"Our long-term hope is to produce a permanent treatment in patients, whether mice, dogs, or humans," Wolfe said.

Named after William Sly of St. Louis University, who discovered the human disorder in 1973, Sly Disease affects many body tissues, including the bone marrow, joints, heart tissue, liver, spleen and brain.

The progressive, debilitating pathology leads to heart and breathing problems, crippling, mental retardation and eventually death within one to two decades.

Although the disease is "individually rare" and affects fewer than 1 out of 100,000 people, it belongs to a broader group of inherited diseases, which together affect 1 in every 1,500 people, said Wolfe.

"All of the diseases have similar strategies for correction," he added.

Potentially, the neural progenitor strategy could be used to treat this wider group of genetic ailments, which include Tay-Sachs, Gaucher's disease, galactosemia and dozens of other maladies that affect millions of people worldwide.

In the future, Snyder and Wolfe plan to attempt the correction of a larger animal model: the dog.

But Wolfe said that because the dog brain is much larger than that of the mouse, it is unclear how much of the dog's particular inherited enzyme-deficiency disease can be corrected.

If the team of researchers are successful yet again, "there will be a strong impetus for us to try our neural progentor technique on humans," said Wolfe.

The first step in developping such a brain-transplant therapy will be to identify and clone neural progenitor cells in humans.

The researchers hope to "do a lot to try to understand the basic cell biology of the neural progenitor cells, the basic forces driving them to become specific cells, and their capabilities from a therapeutic point of view," Snyder said.