Scientists combine therapeutic cloning, embryonic stem cells, and gene defect in mice

While the promise of nuclear transplantation therapy, commonly referred to as "therapeutic cloning," has given hope to patients, like Christopher Reeve, and excited the research community and the public, it has never been successfully demonstrated. Now, scientists from the Whitehead Institute for Biomedical Research have used a mouse model to establish for the first time that a combination of nuclear transplantation, gene therapy, and embryonic stem cell differentiation can be used to create custom-tailored cellular therapies for genetic disorders.

The work is a result of a collaboration between Whitehead Member Rudolf Jaenischs lab and Whitehead Fellow George Daleys lab and will be published as two companion papers on the Cell web site on Friday, March 8, 2002.

The Whitehead researchers joined forces to work on a problem that until now has proven difficult to overcome. Scientists have been able to use nuclear transfer to create embryonic stem cells and differentiate them in culture to create many different cell types, including muscle, neurons, and hematopoietic stem cells, which are the precursors to all immune and blood cells. But, they have never shown that the cells created in culture could be reintroduced into an animal to treat a disease.

Combining their independent research interests, the Jaenisch and Daley labs used skin cells from a mouse, which was completely immune deficient, to create a cellular therapy that was able to partially restore immune function in the mouse. "Though the immune system wasnt completely restored, there was enough improvement to predict that a comparable result in humans would translate into a significant clinical benefit," says Daley.

"This is a proof-of-principle experiment, which shows that nuclear transplantation therapy may be possible for human application. Furthermore, it shows that gene therapy can be incorporated into the approach to correct genetic mutations in defective cells without af

Contact: Nadia Halim
Whitehead Institute for Biomedical Research

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