Realizing the potential of gene therapy for neurological disorders

Promising findings from preclinical animal studies show the potential of gene therapy for treating incurable neurological disorders. Scientists have successfully used gene therapy to slow the progression and improve symptoms of disorders such as amyotrophic lateral sclerosis and Parkinson’s disease.
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Reanalyzing gene tests prompt new diagnoses in kids

A new study quantifies for the first time how quickly rapid advancements in genomics may benefit patients. The research includes a five-year review of more than 300 epilepsy cases that showed about a third of children had a change in diagnosis based on new data. In some cases, the review helped doctors prescribe a more effective treatment.
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Gene that regulates fat accumulation and obesity

A new study showed that regardless of diet, a protein called Pannexin 1 significantly regulates the accumulation of fat in mice. The study suggests that a deletion of the Panx1 gene in the early stages of development of mouse fat cells increases the amount of fat accumulated, leading to a higher risk for obesity later in life.
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New gene therapy reprograms brain glial cells into neurons

A new gene therapy can turn certain brain glial cells into functioning neurons, which in turn could help repair the brain after a stroke or during neurological disorders like Alzheimer’s or Parkinson’s diseases.
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Drug design: Clues for drugging the ‘undruggable’

Scientists have shown how thalidomide analogs mediate degradation of many more proteins than previously anticipated. These proteins — zinc finger transcription factors — play a role in cancer and developmental diseases but are difficult drug targets. The new study suggests that thalidomide analogs can be rationally designed to target this challenging class of proteins.
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First-of-its-kind research models immune responses in cellular immunotherapies

Scientists are pursuing a cross-collaborative effort that could potentially change the way cellular immunotherapies such as stem cell transplantation and CAR T-cell therapies are performed.
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Largest parasitic worm genetic study hatches novel treatment possibilities

The largest genomic study of parasitic worms to date identified hundreds of thousands of new genes and predicted many new potential drug targets and drugs. Research will help scientists understand how these parasites invade, evade the immune system and cause disease.
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