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Letter from the Editor | Ed. 237

The end of a mystery

Published in the December 2010 issue of Pesquisa FAPESP was a report entitled, “The mystery of Ringo and Suflair,” about two golden retrievers, sire and pup, respectively, who had been subjects of a study by researchers at the University of São Paulo (USP). Although the dogs did not produce dystrophin, an essential protein known for maintaining muscle integrity, they did not appear adversely affected.  The researchers were intrigued by this fact and wanted to know what it was that made the dogs practically immune to the problem.  It is this information that might help scientists develop new treatments for people unable to produce dystrophin. After five years and a great deal of research, the USP team – working with researchers from two U.S. centers – found what is known as the Jagged1 gene, located on chromosome 24 in dogs. It was the expression (activation) of this gene that protected the animals. Mystery solved.

But there is a long road ahead before this discovery can be turned into a therapy to help people with forms of muscular dystrophy such as Duchenne and de Becker. New treatments will only be possible if researchers are able to achieve increased activation of the Jagged1 gene – it is the anomaly in this gene that mitigates the effects of the absence of dystrophin.  One important part of the question has already been answered.  What’s needed now are more years of research to obtain tangible results for humans.

The discovery surrounding dystrophin will certainly be useful to precision medicine, an approach that integrates both clinical and molecular data on diseases to come up with treatments tailored to individual patients. Today in São Paulo there are several high-impact initiatives working towards that goal.  The most recent is a shared platform of genetic data called the Brazilian Initiative on Precision Medicine (BIPMed), which will bring together research centers in São Paulo to promote the creation of treatments using genomic medicine.  The São Paulo effort is bringing together the work of five of the 17 Research, Innovation and Dissemination Centers (RIDC) funded by FAPESP. Another important study in this area is a new generation of laboratory tests that can detect cancer at its earliest stages and help assess treatment effectiveness.

Investments in knowledge are seemingly assets that forever reap rewards in every field, even when it takes a long time. In June 2015, a study presented by University of Toronto (Canada) president Méric Gertler underscored this notion. He showed that the São Paulo Metropolitan Region, including the cities of Campinas and São José dos Campos, is one of the urban clusters in which knowledge generated by its universities grew the most from 1996 to 2013, behind only the megalopolises of Shanghai and Beijing, in China, and Seoul in South Korea. According to Gertler, the universities multiplied the number of interactions with companies and organizations in their societies, causing very positive changes in the economy and environment of those cities.

Gertler analyzed the past 20 years’ scientific production and created two rankings. In the first, São Paulo saw a 400% increase in scientific output and now ranks 4th behind only the Asian countries.  The second ranking places the São Paulo cluster in 32nd place based on the volume of scientific production by its institutions between 2011 and 2013.  The study confirms the principle that research universities are growth catalysts for their home regions, boosting innovation, creativity and as a result, the economy.

Pleasant reading.