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Article |
AAV-mediated FOXG1 gene editing in human Rett primary cells
- Susanna Croci
- , Miriam Lucia Carriero
- & Ilaria Meloni
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Article |
High rate of HDR in gene editing of p.(Thr158Met) MECP2 mutational hotspot
- Susanna Croci
- , Miriam Lucia Carriero
- & Ilaria Meloni
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Article |
Cryptic exon activation causes dystrophinopathy in two Chinese families
- Jin Ming
- , Li Jin-Jing
- & Wang Zhi-Qiang
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News and Commentary |
Can manipulation of splicing offer gene therapy possibilities to those with tumour-prone disorders?
- D Gareth R Evans
- , Andrew Wallace
- & William Newman
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Article
| Open AccessIntravitreal delivery of AAV-NDI1 provides functional benefit in a murine model of Leber hereditary optic neuropathy
- Naomi Chadderton
- , Arpad Palfi
- & G Jane Farrar