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5 Results for 'Other therapeutic technologies'
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Technology
COMPOSITIONS AND METHODS FOR PREVENTION OF BLADDER FIBROSIS
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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Dale Bjorling, Jianghui Hou, Zunyi Wang | P200076US02
Technology
REGULATORY ELEMENTS FOR SCHWANN CELL-SPECIFIC GENE EXPRESSION
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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John Svaren | P200262WO01
Technology
Compounds and Methods for Modulating Frataxin Expression in Friedreich’s Ataxia
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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Aseem Ansari, Graham Erwin, Matthew Grieshop | P160232US07
Technology
Gene Correction of Pompe Disease and Other Autosomal Recessive Disorders via CRISPR and other RNA-Guided Nucleases
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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Krishanu Saha, Jared Carlson-Stevermer, Lucille Kohlenberg | P190021US02
Technology
Compounds And Methods For Modulating Frataxin Expression
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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Aseem Ansari, Graham Erwin, Matthew Grieshop | P160232US05