A composition for the treatment of bladder fibrosis in a patient in need that includes a miR-29 mimic is disclosed. The miR-29 mimic may include a working RNA strand with the nucleotide sequence UAGCA...
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Regulatory elements that drive Schwann cell-specific gene expression, nucleic acid vectors, virus particles, and therapeutic compositions incorporating these constructs; and methods of using these var...
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Friedreich’s Ataxia (FRDA) is a neurodegenerative disorder without treatments to halt or reverse disease progression, affecting roughly 9,000 Americans. FRDA is caused by triplet expansion in the fi...
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Pompe disease is a rare autosomal recessive genetic disease (mutations from both parent carriers have to be inherited by the offspring to see symptoms) that typically affects children during developme...
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Friedreich’s ataxia (also referred to as FA or FRDA) is a rare but fatal autosomal recessive neurodegenerative disease. With an estimated incidence of 1 in every 40,000 people, it causes progressive...
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