In vivo base editing of post-mitotic sensory cells
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DOI: 10.1038/s41467-018-04580-3
Abstract: Programmable nucleases can introduce precise changes to genomic DNA through homology-directed repair (HDR). Unfortunately, HDR is largely restricted to mitotic cells, and is typically accompanied by an excess of stochastic insertions and deletions (indels). Here… read more here.
Keywords: base editing; editing post; post mitotic; base ... See more keywords
Efficient precise in vivo base editing in adult dystrophic mice
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DOI: 10.1038/s41467-021-23996-y
Abstract: Recent advances in base editing have created an exciting opportunity to precisely correct disease-causing mutations. However, the large size of base editors and their inherited off-target activities pose challenges for in vivo base editing. Moreover,… read more here.
Keywords: base editing; adult; iabe nga; base ... See more keywords
In vivo base editing by a single i.v. vector injection for treatment of hemoglobinopathies
Sign Up to like & getrecommendations! Published in 2022 at "JCI Insight"
DOI: 10.1172/jci.insight.162939
Abstract: Individuals with β-thalassemia or sickle cell disease and hereditary persistence of fetal hemoglobin (HPFH) possessing 30% fetal hemoglobin (HbF) appear to be symptom free. Here, we used a nonintegrating HDAd5/35++ vector expressing a highly efficient… read more here.
Keywords: editing single; vector; base editing; vivo base ... See more keywords