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rAAV-PHP.B escapes the mouse eye and causes lethality whereas rAAV9 can transduce aniridic corneal limbal stem cells without lethality.
Gene Ther. 2023 Sep;30(9):670-684. doi: 10.1038/s41434-023-00400-6. Epub 2023 Apr 19.
Gene Ther. 2023.
PMID: 37072572
Free PMC article.
LNP-mediated delivery of CRISPR RNP for wide-spread in vivo genome editing in mouse cornea.
Mirjalili Mohanna SZ, Djaksigulova D, Hill AM, Wagner PK, Simpson EM, Leavitt BR.
Mirjalili Mohanna SZ, et al.
J Control Release. 2022 Oct;350:401-413. doi: 10.1016/j.jconrel.2022.08.042. Epub 2022 Aug 27.
J Control Release. 2022.
PMID: 36029893
Free article.
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Germline CRISPR/Cas9-Mediated Gene Editing Prevents Vision Loss in a Novel Mouse Model of Aniridia.
Mirjalili Mohanna SZ, Hickmott JW, Lam SL, Chiu NY, Lengyell TC, Tam BM, Moritz OL, Simpson EM.
Mirjalili Mohanna SZ, et al.
Mol Ther Methods Clin Dev. 2020 Mar 14;17:478-490. doi: 10.1016/j.omtm.2020.03.002. eCollection 2020 Jun 12.
Mol Ther Methods Clin Dev. 2020.
PMID: 32258211
Free PMC article.
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