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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.
Mechanistic investigations of test article-induced pancreatic toxicity at the endocrine-exocrine interface in the rat.
Brenneman KA, Ramaiah SK, Rohde CM, Messing DM, O'Neil SP, Gauthier LM, Stewart ZS, Mantena SR, Shevlin KM, Leonard CG, Sokolowski SA, Lin H, Carraher DC, Jesson MI, Tomlinson L, Zhan Y, Bobrowski WF, Bailey SA, Vogel WM, Morris DL, Whiteley LO, Davis JW 2nd. Brenneman KA, et al. Among authors: whiteley lo. Toxicol Pathol. 2014 Jan;42(1):229-42. doi: 10.1177/0192623313508851. Epub 2013 Nov 13. Toxicol Pathol. 2014. PMID: 24226507
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.
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.
Nonclinical Studies that Support Viral Vector-Delivered Gene Therapies: An EFPIA Gene Therapy Working Group Perspective.
Bolt MW, Whiteley LO, Lynch JL, Lauritzen B, Fernández de Henestrosa AR, MacLachlan T, Ulrich P, Philip BK, Mahalingaiah PK, Fuller CL, Compton DR. Bolt MW, et al. Among authors: whiteley lo. Mol Ther Methods Clin Dev. 2020 Sep 1;19:89-98. doi: 10.1016/j.omtm.2020.08.017. eCollection 2020 Dec 11. Mol Ther Methods Clin Dev. 2020. PMID: 33024793 Free PMC article.
Comparison of hepatic transcription profiles of locked ribonucleic acid antisense oligonucleotides: evidence of distinct pathways contributing to non-target mediated toxicity in mice.
Kakiuchi-Kiyota S, Koza-Taylor PH, Mantena SR, Nelms LF, Enayetallah AE, Hollingshead BD, Burdick AD, Reed LA, Warneke JA, Whiteley LO, Ryan AM, Mathialagan N. Kakiuchi-Kiyota S, et al. Among authors: whiteley lo. Toxicol Sci. 2014 Mar;138(1):234-48. doi: 10.1093/toxsci/kft278. Epub 2013 Dec 11. Toxicol Sci. 2014. PMID: 24336348
56 results