相关实验视频

Updated: Jan 14, 2026

Surgical Method for Virally Mediated Gene Delivery to the Mouse Inner Ear through the Round Window Membrane

Surgical Method for Virally Mediated Gene Delivery to the Mouse Inner Ear through the Round Window Membrane

Published on: March 16, 2015

关于遗传性听力损失的基因治疗的国际专家共识:基于临床试验

Xintai Fan¹, Ziwen Gao¹, Jiake Zhong²

¹ENT Institute and Department of Otorhinolaryngology, Eye & ENT Hospital, Fudan University, Shanghai, China; Shanghai Key Laboratory of Gene Editing and Cell Therapy for Rare Diseases, Fudan University, Shanghai, China; NHC Key Laboratory of Hearing Medicine, Shanghai, China.

Med (New York, N.Y.)

|October 24, 2025

概括

这项研究为遗传性听力损失的基因疗法建立了第一个国际共识,为临床试验和患者护理创造了标准化的框架. 这些指导方针旨在加速研究翻译,并确保新兴治疗方法的安全性.

科学领域:

耳鼻喉科耳鼻喉科耳鼻喉科
遗传学是一个遗传学.
听力学听力学是指听力学.
基因治疗基因治疗

背景情况:

遗传性听力损失是一种常见的儿童疾病,治疗选择有限.
在OTOF基因疗法的进步突出了标准化临床指导方针的需要.
这项研究解决了关于遗传性听力损失的基因治疗的国际共识的迫切需要.

研究的目的:

建立第一个关于遗传性听力损失基因疗法的临床应用的国际共识.
为儿童听力损失中新兴基因疗法提供标准化框架.

主要方法:

一个经过修改的Delphi过程,涉及46名多学科专家,从2024年3月到2025年3月.
系统的文献审查和专家知识的整合.
三轮代投票,包括匿名调查和在线共识会议,声明需要≥75%的同意.

主要成果:

在六个领域制定了30份共识声明:伦理审查,患者选择,诊断,药物输送,随访和康复.
通过对最初的9,093.3个出版物中的69个出版物的审查来支持声明.
关键领域包括详细的患者选择标准和基因治疗的手术前评估.

结论:

这一共识建立了第一个全球认可的基因疗法在遗传性听力损失的框架.
这些指导方针标准化了临床试验设计和患者管理,促进了安全性和加速了研究翻译.
该框架适用于OTOF相关的听力损失,并适应其他遗传形式的听力损失.

关键词:

翻译为人口健康健康.腺相关病毒的病毒.临床试验临床试验临床试验临床试验临床试验专家的共识专家的共识基因治疗的基因疗法遗传性听力损失是一种遗传性听力损失.修改后的德尔菲方法.

更多相关视频

Posterior Semicircular Canal Approach for Inner Ear Gene Delivery in Neonatal Mouse

Posterior Semicircular Canal Approach for Inner Ear Gene Delivery in Neonatal Mouse

Published on: March 2, 2018

Canalostomy As a Surgical Approach to Local Drug Delivery into the Inner Ears of Adult and Neonatal Mice

Canalostomy As a Surgical Approach to Local Drug Delivery into the Inner Ears of Adult and Neonatal Mice

Published on: May 25, 2018

相关概念视频

Gene Therapy

Gene Therapy

Gene therapy is a technique where a gene is inserted into a person’s cells to prevent or treat a serious disease. The added gene may be a healthy version of the gene that is mutated in the patient, or it could be a different gene that inactivates or compensates for the patient’s disease-causing gene. For example, in patients with severe combined immunodeficiency (SCID) due to a mutation in the gene for the enzyme adenosine deaminase, a functioning version of the gene can be...

Hearing

Hearing

When we hear a sound, our nervous system is detecting sound waves—pressure waves of mechanical energy traveling through a medium. The frequency of the wave is perceived as pitch, while the amplitude is perceived as loudness.

What is Genetic Engineering?

What is Genetic Engineering?