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Otoferlin gene-mediated hearing loss is a sensorineural hearing loss
Gene therapy for ear diseases
Ear diseases, mostly manifested as deafness, also known as hearing impairment, are one ofFrench scientists used two different recombinant AAV vectors through a strategy called double AAV, one carrying a 5'-fragment of otoferlin cDNA (OTOF NT, nucleotide 1-2,448) and the other carrying a 3'-fragment (OTOF CT, nucleotide 2,449-5,979), both of which are capable of repeated recombination through their reverse terminals and producing polypsies, and the resulting transcript can be properly spliced to produce the protein
Ruqian Liu's team successfully restored the hearing of deaf mice mutated by Tmc1 gene by using dual AAV vector delivery technology and the repair of mutated genes by new single-base gene editing technology, and published relevant research
epilogue
Gene therapies are bringing new hope to a growing number of disease fields, especially for genetic diseases with genetic defectsResources:
1.
——List of recent hot events——
▼September 20, CAR-T cell drug commercial development strategy▼September 22, Efficient synthesis strategy
and process flow analysis of oligonucleotide drugs
▼On September 21, Merck China Biosafety Testing Center was opened
