新型基因療法成功逆轉先天性耳聾
New Gene Therapy Successfully Reverses Congenital Deafness
一項開創性的醫學里程碑已經達成:基因療法目前已成功逆轉了先天性耳聾患者因 OTOF 基因相關引起的聽力損失。
A groundbreaking medical milestone has arrived: gene therapy is now successfully reversing congenital deafness in patients with OTOF-related hearing loss.
這種病症導致人體無法製造耳畸蛋白,這是將聲音傳輸至大腦所需的蛋白質。
This condition prevents the body from producing otoferlin, a protein necessary for transmitting sound to the brain.
研究人員利用一種無害病毒將具備功能的 OTOF 基因直接送入內耳,使細胞得以製造缺失的蛋白質。
Researchers use a harmless virus to deliver a functional OTOF gene directly into the inner ear, allowing cells to produce the missing protein.
與使用電子的耳蝸植入物不同,這種療法修復的是生物機制本身。
Unlike cochlear implants that use electronics, this therapy repairs the biological machinery itself.
包含 CHORD 研究在內的試驗顯示,此程序即使在年齡較大的兒童與成人身上,也既安全又有效。
The trials, including the CHORD study, show the procedure is safe and effective even in older children and adults.
雖然目前聚焦於 OTOF 突變,但這項成功為治癒其他類型的遺傳性聽力損失提供了藍圖,並帶來希望,許多過去被認為是永久性的病症,或許不久後將能獲得治療。
While currently focused on OTOF mutations, this success serves as a blueprint for curing other forms of genetic hearing loss, offering hope that many previously permanent conditions may soon be treatable.
