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Next Decade Of Brain Research Promises New Answers

November 2, 2010

In the decade since the first draft sequence of the human genome was released, a new field ““ neurogenetics ““ has taken wing, said leaders in the field from Baylor College of Medicine and Emory University School of Medicine in Atlanta in an overview published in the journal Neuron.

It was already building toward such success. In the 1990s, using the tools available at the time, scientists had already found critical genes such as that for Duchenne muscular dystrophy, Charcot-Marie Tooth and fragile X, said Dr. Huda Y. Zoghbi, professor of molecular and human genetics, pediatrics, neuroscience and neurology at BCM, and Dr. Stephen T. Warren, professor of human genetics, pediatrics and biochemistry at Emory. Zoghbi is also director of the Jan and Dan Duncan Neurological Research Institute at Texas Children’s Hospital and a Howard Hughes Medical Institute investigator.
Mapping and sequencing

Zoghbi and her colleagues at BCM cloned the gene for Rett syndrome in 1999, a success made possible by the intense mapping and sequencing efforts of the X chromosome that preceded it. In their essay, Zoghbi and Warren note that national investment in such research has already paid off, not only in diagnosis and understanding of such diseases but also in the outlook for families afflicted by the disorders.

“Today, a large number of childhood and adult neurological disorders can be diagnosed by a simple DNA test on peripheral blood,” the authors wrote. It is also easier to provide families with information on their risk of having another child with the disorder.
Therapy development slow

“While arguably the pace of development of potential therapies has been relatively slow compared to the speed of disease gene discovery, we should not underestimate the great benefits to families of disease prevention through prenatal diagnosis, and the gains in fundamental neurobiology from pathogenesis studies of neurological disorders,” the authors wrote.

The genomes of model animal systems have also been sequenced, preparing the way for studies of treatments based on new understanding of neurobiology. When such work is ready for human testing, they note that it requires an appropriate infrastructure built on partnerships among academic research, government, private institutions, foundations and the pharmaceutical industry.
Principles for the future

The future will be enhanced by following a few principles:

    * Invest in studies that will aid better understanding of normal brain development, as this will help disorders afflicting children as well as the adult and aging brain.
    * The role of epigenetics (factors that affect the ability of genes to carry out their functions without altering their DNA sequence) and how it governs disease development must be carefully studied with an eye to modulating its effects to subdue disease.
    * Understanding the plasticity and resilience of the developing and adult brain will help map the future of treatment and care.

“The findings that several disorders, including some of the more devastating developmental and degenerative diseases are reversible in mouse models, provides hope that discovering ways to counteract or suppress diseases might halt or even reverse some of the most serious neurological and psychiatric disorders,” they wrote.

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