PMC · DOI:10.1016/j.gendis.2025.101928·November 8, 2025
In vivo CRISPR/Cas9-mediated gene integration corrects mucopolysaccharidosis type II in mice
Hanfei Yu, Qian Qin, Shiyu Cui, Yujuan Wa, Kexian Dong, Wei Ji, Xueyuan Jia, Songbin Fu, Jie Wu, Wenjing Sun
Abstract
Linked entities
Genes, proteins, chemicals, diseases, species, mutations and cell lines named across the full text — each resolved to its canonical identifier and authoritative record.
Diseases1
Figures1
Click any figure to enlarge with its caption.
Figure 1Peer Reviews
No public reviews on file for this paper yet. If you reviewed it on a platform where reviews are public (OpenReview, ICLR, NeurIPS, ICML), you can paste yours below so the community can read it here.
Videos
No videos yet. Explain this paper in a talk, walkthrough, or lecture? Add one.
Taxonomy
TopicsLysosomal Storage Disorders Research · CRISPR and Genetic Engineering · Glycogen Storage Diseases and Myoclonus