Technology news and Jobs arrow VIRTUALISATION arrow Can we cure diseases? We've leaped forward!
Can we cure diseases? We've leaped forward! E-mail
by William Atkins   
Monday, 01 June 2009
Lead by scientists at the Salk Institute for Biological Studies, a team of researchers has successfully shown that, in theory, human diseases can be cured using gene therapy and stem cells. There’s much more work to accomplish, but this research is highly important for eventually curing cancers and other such diseases.


The Salk Institute team corrected a defective gene in cells removed from patients with the genetic disease Fanconi anemia, which often produces short stature and skeletal problems, along with eventually leukemia, bone marrow failure, and other forms of cancer.

Their technique involves taking skin or hair cells from patients with the Fanconi anemia disease.

They, then, use several procedures to remove the gene defect and reprogram the cells into induced pluripotent stem cells (iPS cells, or iPSCs), which are a type of stem cells artificially derived (usually) from an adult somatic cell by forcing certain genes to change.

Ángel Raya, Ignasi Rodríguez-Pizà, Guillermo Guenechea, Rita Vassena, Susana Navarro, María José Barrero, Antonella Consiglio, Maria Castellà, Paula Río, Eduard Sleep, Federico González Gustavo Tiscornia Elena Garreta, Trond Aasen, Anna Veiga Inder M. Verma, Jordi Surrallés, Juan Bueren, and Juan Carlos Izpisúa Belmonte wrote in the abstract to their paper: “The generation of induced pluripotent stem (iPS) cells has enabled the derivation of patient-specific pluripotent cells and provided valuable experimental platforms to model human disease. Patient-specific iPS cells are also thought to hold great therapeutic potential, although direct evidence for this is still lacking.”

The paper “Disease-corrected haematopoietic progenitors from Fanconi anaemia induced pluripotent stem cells” appears online May 31, 2009, in the journal Nature (doi:10.1038/nature08129).

According to the June 1, 2009 ScienceDaily.com article Combined Stem Cell-Gene Therapy Approach Cures Human Genetic Disease In Vitro, “The study is a major milestone on the path from the laboratory to the clinic.”

The authors state in their abstract (to their paper), “… we show that, on correction of the genetic defect, somatic cells from Fanconi anaemia patients can be reprogrammed to pluripotency to generate patient-specific iPS cells. These cell lines appear indistinguishable from human embryonic stem cells and iPS cells from healthy individuals."

Page two continues.



 
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