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Promoterless, Nuclease-Free Genome Editing Confers a Growth Advantage for Corrected Hepatocytes in Mice With Methylmalonic Acidemia.
Hepatology. 2021 Jun;73(6):2223-2237. doi: 10.1002/hep.31570. Epub 2021 May 21.
Hepatology. 2021.
PMID: 32976669
Free PMC article.
Treatment of metabolic disorders using genomic technologies: Lessons from methylmalonic acidemia.
Venturoni LE, Venditti CP.
Venturoni LE, et al.
J Inherit Metab Dis. 2022 Sep;45(5):872-888. doi: 10.1002/jimd.12534. Epub 2022 Jul 21.
J Inherit Metab Dis. 2022.
PMID: 35766386
Review.
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Growth advantage of corrected hepatocytes in a juvenile model of methylmalonic acidemia following liver directed adeno-associated viral mediated nuclease-free genome editing.
Venturoni LE, Chandler RJ, Liao J, Hoffmann V, Ramesh N, Gordo S, Chau N, Venditti CP.
Venturoni LE, et al.
Mol Genet Metab. 2022 Sep-Oct;137(1-2):1-8. doi: 10.1016/j.ymgme.2022.06.011. Epub 2022 Jul 4.
Mol Genet Metab. 2022.
PMID: 35868241
Free PMC article.
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Central nervous system-targeted adeno-associated virus gene therapy in methylmalonic acidemia.
May FJ, Head PE, Venturoni LE, Chandler RJ, Venditti CP.
May FJ, et al. Among authors: venturoni le.
Mol Ther Methods Clin Dev. 2021 Jun 3;21:765-776. doi: 10.1016/j.omtm.2021.04.005. eCollection 2021 Jun 11.
Mol Ther Methods Clin Dev. 2021.
PMID: 34169115
Free PMC article.
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In vivo genome editing at the albumin locus to treat methylmalonic acidemia.
Schneller JL, Lee CM, Venturoni LE, Chandler RJ, Li A, Myung S, Cradick TJ, Hurley AE, Lagor WR, Bao G, Venditti CP.
Schneller JL, et al. Among authors: venturoni le.
Mol Ther Methods Clin Dev. 2021 Nov 11;23:619-632. doi: 10.1016/j.omtm.2021.11.004. eCollection 2021 Dec 10.
Mol Ther Methods Clin Dev. 2021.
PMID: 34901307
Free PMC article.
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