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A modified mouse model of Friedreich's ataxia with conditional Fxn allele homozygosity delays onset of cardiomyopathy.
Perfitt TL, Huichalaf C, Gooch R, Kuperman A, Ahn Y, Chen X, Ullas S, Hirenallur-Shanthappa D, Zhan Y, Otis D, Whiteley LO, Bulawa C, Martelli A. Perfitt TL, et al. Among authors: whiteley lo. Am J Physiol Heart Circ Physiol. 2024 Feb 1;326(2):H357-H369. doi: 10.1152/ajpheart.00496.2023. Epub 2023 Dec 1. Am J Physiol Heart Circ Physiol. 2024. PMID: 38038720
In vivo overexpression of frataxin causes toxicity mediated by iron-sulfur cluster deficiency.
Huichalaf C, Perfitt TL, Kuperman A, Gooch R, Kovi RC, Brenneman KA, Chen X, Hirenallur-Shanthappa D, Ma T, Assaf BT, Pardo I, Franks T, Monarski L, Cheng TW, Le K, Su C, Somanathan S, Whiteley LO, Bulawa C, Pregel MJ, Martelli A. Huichalaf C, et al. Among authors: whiteley lo. Mol Ther Methods Clin Dev. 2022 Feb 7;24:367-378. doi: 10.1016/j.omtm.2022.02.002. eCollection 2022 Mar 10. Mol Ther Methods Clin Dev. 2022. PMID: 35252470 Free PMC article.
Biodistribution and Tolerability of AAV-PHP.B-CBh-SMN1 in Wistar Han Rats and Cynomolgus Macaques Reveal Different Toxicologic Profiles.
Palazzi X, Pardo ID, Sirivelu MP, Newman L, Kumpf SW, Qian J, Franks T, Lopes S, Liu J, Monarski L, Casinghino S, Ritenour C, Ritenour H, Dubois C, Olson J, Graves J, Alexander KE, Coskran T, Lanz TA, Brady J, McCarty D, Suryanarayan S, Whiteley LO. Palazzi X, et al. Among authors: whiteley lo. Hum Gene Ther. 2022 Feb;33(3-4):175-187. doi: 10.1089/hum.2021.116. Hum Gene Ther. 2022. PMID: 34931542 Free PMC article.
Scientific and Regulatory Policy Committee Points to Consider: Nonclinical Research and Development of In Vivo Gene Therapy Products, Emphasizing Adeno-Associated Virus Vectors.
Hutt JA, Assaf BT, Bolon B, Cavagnaro J, Galbreath E, Grubor B, Kattenhorn LM, Romeike A, Whiteley LO. Hutt JA, et al. Among authors: whiteley lo. Toxicol Pathol. 2022 Jan;50(1):118-146. doi: 10.1177/01926233211041962. Epub 2021 Oct 17. Toxicol Pathol. 2022. PMID: 34657529 Review.
Zucker Lean Rats With Hepatic Steatosis Recapitulate Asymptomatic Metabolic Syndrome and Exhibit Greater Sensitivity to Drug-Induced Liver Injury Compared With Standard Nonclinical Sprague-Dawley Rat Model.
LaBranche TP, Kopec AK, Mantena SR, Hollingshead BD, Harrington AW, Stewart ZS, Zhan Y, Hayes KD, Whiteley LO, Burdick AD, Davis JW 2nd. LaBranche TP, et al. Among authors: whiteley lo. Toxicol Pathol. 2020 Dec;48(8):994-1007. doi: 10.1177/0192623320968716. Epub 2020 Nov 28. Toxicol Pathol. 2020. PMID: 33252024
52 results