Stem-Cell Therapy Successful in Altering Defective Gene

A team of researchers has announced combined stem cell-gene therapy that has corrected a defective gene in cells taken from patients with a disorder called Fanconi Anemia. The study was published on May 31, 2009 in the magazine Nature.

The team of researchers have collaborated from several institutions, including the Universitat Autònoma de Barcelona, and the Centre for Regenerative Medicine of  Barcelona, CIEMAT in Madrid, and The Salk Institute for Bilogical Studies in La Jolla, Ca.  Dr. Juan Carlos Izpisua Belmonte, a professor at the Centre for Regenerative Medicine of Barcelona comments, “We haven’t cured a human being, but we have cured a cell. In theory we could transplant it into a human and cure the disease.”

Dr. Belmonte continues, “The hope in the field has always been that we’ll be able to correct a disease genetically and then make iPS cells that differentiate into the type of tissue where the disease is manifested and bring it to clinic.”

In this study, hair and or skin cells were taken from a patient with Fanconi anemia.  The genetic defect in this cell is converted to induced pluripotent stem cells or iPS cells , which are similar to embryonic stem cells.

Fanconi anemia is a genetic disorder that can lead to bone marrow failure and cancers such as leukemia.

The techniue is not perfected and a significant challenge is to work on preventing mutations which lead to tumors.

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