VESTAL, N.Y. — A Binghamton University professor will use grant funding of $329,000 for research focused on Duchenne muscular dystrophy (DMD).
Kanneboyina Nagaraju, professor and chair of the department of pharmaceutical sciences, and his team at Binghamton University’s School of Pharmacy and Pharmaceutical Sciences will use the funding, the school said in a news release.
Hackensack, New Jersey–based Parent Project Muscular Dystrophy (PPMD), a nonprofit organization that says it is “leading the fight to end DMD,” awarded the funding.
With the monetary assistance, Nagaraju will continue his work examining the body’s immune response to the production of new dystrophin protein resulting from “exon skipping” and gene-therapy treatments.
Duchenne is the most common fatal genetic disorder diagnosed in childhood, affecting about one in 5,000 live male births.
Caused by a change in the dystrophin gene, the disease robs muscles of the ability to function or repair themselves properly, per the release.
Duchenne currently has no cure, but researchers are pursuing both exon skipping and gene therapy as potential treatments that may slow its progression. As the name implies, the goal of exon skipping is to encourage the cellular machinery to “skip over” an exon that has been deleted by Duchenne muscular dystrophy and interfered with the rest of the gene’s development, according to the website of Muscular Dystrophy UK.
Binghamton University says Nagaraju hopes to understand the effect these treatments may have on the body’s immune system.
“Long-term immunological consequences of dystrophin gene correction and gene-replacement therapeutic strategies in DMD are currently unknown,” Nagaraju said. “This project will explore interventions that modulate inflammation and immune response after gene and exon skipping therapies. I am really excited that PPMD recognized the merits of the studies proposed in this grant.”
PPMD has funded research aimed at treating Duchenne for almost 25 years and “finally” therapies like exon skipping and gene therapy are “near term.” Abby Bronson, PPMD’s senior VP of research strategy, said in the Binghamton news release.
“Now we need to look ahead to optimize the effectiveness of these promising new therapies,” said Bronson. “We believe there is great value in the work Dr. Nagaraju and his team are doing — work that could improve the post-treatment outcome of these potential therapies.”
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