Cutting through the stones: Unlocking therapeutic potential with gene editing tools for primary hyperoxaluria type 1.
作者信息
Schneller Jessica, Du Wan, Ding Hui
机构信息
The Vector, American Society of Gene & Cell Therapy, Waukesha, WI, USA.
Harvard Medical School, Boston, MA, USA; Mass General Brigham, Boston, MA, USA.
出版信息
Mol Ther. 2025 Jan 8;33(1):11-13. doi: 10.1016/j.ymthe.2024.12.030. Epub 2024 Dec 24.
Abstract
摘要
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本文引用的文献
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Efficient and safe in vivo treatment of primary hyperoxaluria type 1 via LNP-CRISPR-Cas9-mediated glycolate oxidase disruption.通过LNP-CRISPR-Cas9介导的乙醇酸氧化酶破坏对1型原发性高草酸尿症进行高效安全的体内治疗。
Mol Ther. 2025 Jan 8;33(1):104-118. doi: 10.1016/j.ymthe.2024.10.003. Epub 2024 Oct 9.
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Efficient and safe therapeutic use of paired Cas9-nickases for primary hyperoxaluria type 1.高效、安全的 Cas9 内切酶-尼克酰胺酶对 1 型原发性高草酸尿症的治疗作用。
EMBO Mol Med. 2024 Jan;16(1):112-131. doi: 10.1038/s44321-023-00008-8. Epub 2024 Jan 5.
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