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Updated: Mar 2, 2026

Efficient PAM-Less Base Editing for Zebrafish Modeling of Human Genetic Disease with zSpRY-ABE8e
07:31

Efficient PAM-Less Base Editing for Zebrafish Modeling of Human Genetic Disease with zSpRY-ABE8e

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精密かつ効率的なDNA塩基編集により、成人DFNB9マウスモデルで正常な聴覚を回復

Ziyu Zhang1, Man Wang1, Fangzhi Tan1

  • 1Department of Otolaryngology Head and Neck Surgery, Zhongda Hospital, State Key Laboratory of Digital Medical Engineering, Jiangsu Provincial Key Laboratory of Critical Care Medicine, School of Life Sciences and Technology, School of Medicine, Advanced Institute for Life and Health, Southeast University, Nanjing 210096, China.

Med (New York, N.Y.)
|February 28, 2026
PubMed
まとめ

遺伝子編集によりOTOF欠損マウスの聴覚が正常に回復し、DFNB9難聴治療における広範な治療ウィンドウが実証された。このアデニン塩基編集アプローチは、将来の遺伝子治療に有望である。

キーワード:
AAV遺伝子治療DFNB9DNA塩基編集Otof変異蝸牛デュアルAAV聴覚回復遺伝性難聴内耳前臨床研究治療時間ウィンドウ

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In-vitro Mutagenesis01:16

In-vitro Mutagenesis

To learn more about the function of a gene, researchers can observe what happens when the gene is inactivated or “knocked out,” by creating genetically engineered knockout animals. Knockout mice have been particularly useful as models for human diseases such as cancer, Parkinson’s disease, and diabetes.
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