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METHODS OF TREATING GENETIC HEARING LOSS
专利权人:
UNIVERSITY OF IOWA RESEARCH FOUNDATION
发明人:
Richard J. SMITH,Paul T. RANUM
申请号:
US16635422
公开号:
US20200248204A1
申请日:
2018.08.02
申请国别(地区):
US
年份:
2020
代理人:
摘要:
In certain embodiments the present invention provides a method of treating hearing loss comprising: (a) administering a gene suppression agent that suppresses both copies of an endogenous gene causing the hearing loss; and (b) administering an exogenous wild-type allele engineered to resist suppression by the gene suppression agent. The present invention provides in certain embodiments a method of treating a genetic hearing loss (GHL) in a patient in need thereof comprising: (a) identifying a mutation in a GHL-causing gene, wherein the mutation causes GHL in the patient; and (b) administering to the patient a pharmaceutical composition comprising a therapeutic miRNA and a pharmaceutically acceptable carrier, wherein the GHL therapeutic miRNA is of 18 to 25 nucleotides in length and knocks-down the GHL-causing gene function at a higher level than it knocks-down gene function in a corresponding wild-type gene.
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