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Archive - Jun 15, 2019

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Researchers' Discovery Could Lead to Improved Therapies for Duchenne Muscular Dystrophy; Administration of Sarcospan Protein May Strengthen Heart

A new multi-institution study spearheaded by researchers at Florida State University (FSU) and the University of California, Los Angeles (UCLA) suggests a tiny protein could play a major role in combating heart failure related to Duchenne muscular dystrophy (DMD), the most common lethal genetic disorder among children. In collaboration with scientists from across the nation, FSU researchers found that increased levels of the protein sarcospan improve cardiac function by reinforcing cardiac cell membranes, which become feeble in patients with DMD. Their findings were published online on April 30, 2019 in JCI Insight. The article is titled “Stabilization of the Cardiac Sarcolemma by Sarcospan Rescues DMD-Associated Cardiomyopathy.” The condition, which typically afflicts young boys, is caused by a mutation that prevents the body from producing dystrophin, a protein crucial to the health of skeletal, respiratory, and cardiac muscles. Advances in treatment for certain types of DMD-related muscle degradation have helped to prolong patients' lifespans. However, as DMD patients age, their heart function declines dramatically. "Patients typically live to 20 or 30 years of age," said lead author Michelle Parvatiyar (photo), PhD, an Assistant Professor in the Department of Nutrition, Food and Exercise Sciences in FSU's College of Human Sciences. "There have been important improvements in respiratory care, which used to be what a majority of patients would succumb to. Now, in their 20s and 30s, they're often succumbing to cardiomyopathy. The heart is functioning with a major component of the cell membrane missing. Over time, it wears out."

Researchers Gain Useful Vaccination Clues from Studying Mother’s Transfer of Antibodies Across Placenta to Fetus

One of the most successful interventions in reducing infectious disease worldwide, vaccination still has limited effectiveness in protecting one group of patients - newborn infants. Now a study based at the Ragon Institute of MGH, MIT and Harvard has determined how a pregnant woman's vaccine-induced immunity is transferred to her child, which has implications for the development of more effective maternal vaccines. The report will be published in the June 27 issue of Cell and, receiving early online release on June 13, 2019. The article is titled “Fc Glycan-Mediated Regulation of Placental Antibody Transfer” "Newborns arrive into the world on the first day of life with brand-new immune systems that, like the children themselves, need to learn to cope with both helpful and harmful microbes in their environment," says Galit Alter (photo), PhD, of the Ragon Institute and the Massachusetts General Hospital (MGH) Department of Medicine, co-senior author of the Cell paper. "To help the newborn immune system learn to discriminate between friend and foe, mothers transfer antibodies to their infants via the placenta. The rules by which the placenta performs this absolutely essential function have been unknown but, if decoded, could hold the key to generating more powerful vaccines to protect these most precious patients." While maternal antibodies against some diseases such as measles can be transferred from mother to infant, providing some protection until the child is old enough for individual vaccination, antibodies to other serious diseases like polio are less efficiently transferred. To investigate the mechanisms by which antibodies are transferred from mother to child, Dr.