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Andrea Frassetto

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Citations 1342
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Recent Articles
1.
Zhou J, Liang S, Yin L, Frassetto A, Graham A, White R, et al.
Mol Ther . 2025 Jan; 33(3):1197-1212. PMID: 39799396
Ornithine transcarbamylase deficiency (OTCD) is the most common urea-cycle disorder, characterized by hyperammonemia and accompanied by a high unmet patient need. mRNA therapies have been shown to be efficacious in...
2.
Touramanidou L, Gurung S, Cozmescu C, Perocheau D, Moulding D, Finn P, et al.
Cells . 2024 Dec; 13(23). PMID: 39682727
Recently approved adeno-associated viral (AAV) vectors for liver monogenic diseases haemophilia A and B are exemplifying the success of liver-directed viral gene therapy. In parallel, additional gene therapy strategies are...
3.
Coughlan K, Eybye M, Henderson N, DeAntonis C, Frassetto A, Hanahoe E, et al.
Mol Genet Metab . 2024 Aug; 143(1-2):108560. PMID: 39121792
Isolated methylmalonic acidemia/aciduria (MMA) due to MMUT enzyme deficiency is an ultra-rare pediatric disease with high morbidity and mortality, with no approved disease-altering therapies. Previous publications showed that systemic treatment...
4.
Chambers C, Soo G, Engel A, Glass I, Frassetto A, Martini P, et al.
Transl Vis Sci Technol . 2024 Jul; 13(7):7. PMID: 38980261
Purpose: Lipid nanoparticles (LNPs) show promise in their ability to introduce mRNA to drive protein expression in specific cell types of the mammalian eye. Here, we examined the ability of...
5.
Baek R, Coughlan K, Jiang L, Liang M, Ci L, Singh H, et al.
Nat Commun . 2024 May; 15(1):3804. PMID: 38714648
Messenger RNA (mRNA) therapeutics delivered via lipid nanoparticles hold the potential to treat metabolic diseases caused by protein deficiency, including propionic acidemia (PA), methylmalonic acidemia (MMA), and phenylketonuria (PKU). Herein...
6.
Perocheau D, Gurung S, Touramanidou L, Duff C, Sharma G, Sebire N, et al.
F1000Res . 2024 Apr; 12:1580. PMID: 38618017
Background: In academic research and the pharmaceutical industry, cell lines and animal models are considered as gold standards in modelling diseases and assessing therapeutic efficacy. However, both models have intrinsic...
7.
Gurung S, Timmermand O, Perocheau D, Gil-Martinez A, Minnion M, Touramanidou L, et al.
Sci Transl Med . 2024 Jan; 16(729):eadh1334. PMID: 38198573
The urea cycle enzyme argininosuccinate lyase (ASL) enables the clearance of neurotoxic ammonia and the biosynthesis of arginine. Patients with ASL deficiency present with argininosuccinic aciduria, an inherited metabolic disease...
8.
Gurung S, Karamched S, Perocheau D, Seunarine K, Baldwin T, Alrashidi H, et al.
J Inherit Metab Dis . 2023 Dec; 47(6):1213-1227. PMID: 38044746
Argininosuccinate lyase (ASL) is integral to the urea cycle detoxifying neurotoxic ammonia and the nitric oxide (NO) biosynthesis cycle. Inherited ASL deficiency causes argininosuccinic aciduria (ASA), a rare disease with...
9.
Chambers C, Soo G, Engel A, Glass I, Frassetto A, Martini P, et al.
bioRxiv . 2023 Jul; PMID: 37502987
Purpose: Lipid nanoparticles (LNPs) show promise in their ability to introduce mRNA to drive protein expression in specific cell types of the mammalian eye. Here, we examined the ability of...
10.
Faccioni F, Bevilacqua L, Porrelli D, Khoury A, Faccioni P, Turco G, et al.
Int J Oral Maxillofac Implants . 2021 Jun; 36(3):520-528. PMID: 34115066
Purpose: To assess the effects of grade IV titanium ultrasonic tip instrumentation on different grade IV titanium implant surfaces and compare the decontamination of different implant surfaces using chlorhexidine, blue...