Buza EL - Search Results

1

Efficacy and Safety of a Krabbe Disease Gene Therapy.

Hordeaux J, Jeffrey BA, Jian J, Choudhury GR, Michalson K, Mitchell TW, Buza EL, Chichester J, Dyer C, Bagel J, Vite CH, Bradbury AM, Wilson JM. Hordeaux J, et al. Among authors: buza el. Hum Gene Ther. 2022 May;33(9-10):499-517. doi: 10.1089/hum.2021.245. Epub 2022 Mar 22. Hum Gene Ther. 2022. PMID: 35333110 Free PMC article.

2

Severe Toxicity in Nonhuman Primates and Piglets Following High-Dose Intravenous Administration of an Adeno-Associated Virus Vector Expressing Human SMN.

Hinderer C, Katz N, Buza EL, Dyer C, Goode T, Bell P, Richman LK, Wilson JM. Hinderer C, et al. Among authors: buza el. Hum Gene Ther. 2018 Mar;29(3):285-298. doi: 10.1089/hum.2018.015. Epub 2018 Feb 12. Hum Gene Ther. 2018. PMID: 29378426 Free PMC article.

3

The Neurotropic Properties of AAV-PHP.B Are Limited to C57BL/6J Mice.

Hordeaux J, Wang Q, Katz N, Buza EL, Bell P, Wilson JM. Hordeaux J, et al. Among authors: buza el. Mol Ther. 2018 Mar 7;26(3):664-668. doi: 10.1016/j.ymthe.2018.01.018. Epub 2018 Feb 2. Mol Ther. 2018. PMID: 29428298 Free PMC article.

4

Meganuclease targeting of PCSK9 in macaque liver leads to stable reduction in serum cholesterol.

Wang L, Smith J, Breton C, Clark P, Zhang J, Ying L, Che Y, Lape J, Bell P, Calcedo R, Buza EL, Saveliev A, Bartsevich VV, He Z, White J, Li M, Jantz D, Wilson JM. Wang L, et al. Among authors: buza el. Nat Biotechnol. 2018 Sep;36(8):717-725. doi: 10.1038/nbt.4182. Epub 2018 Jul 9. Nat Biotechnol. 2018. PMID: 29985478

5

Toxicology Study of Intra-Cisterna Magna Adeno-Associated Virus 9 Expressing Iduronate-2-Sulfatase in Rhesus Macaques.

Hordeaux J, Hinderer C, Goode T, Buza EL, Bell P, Calcedo R, Richman LK, Wilson JM. Hordeaux J, et al. Among authors: buza el. Mol Ther Methods Clin Dev. 2018 Jul 14;10:68-78. doi: 10.1016/j.omtm.2018.06.004. eCollection 2018 Sep 21. Mol Ther Methods Clin Dev. 2018. PMID: 30073178 Free PMC article.

6

Toxicology Study of Intra-Cisterna Magna Adeno-Associated Virus 9 Expressing Human Alpha-L-Iduronidase in Rhesus Macaques.

Hordeaux J, Hinderer C, Goode T, Katz N, Buza EL, Bell P, Calcedo R, Richman LK, Wilson JM. Hordeaux J, et al. Among authors: buza el. Mol Ther Methods Clin Dev. 2018 Jul 14;10:79-88. doi: 10.1016/j.omtm.2018.06.003. eCollection 2018 Sep 21. Mol Ther Methods Clin Dev. 2018. PMID: 30073179 Free PMC article.

7

Adeno-associated viral gene therapy corrects a mouse model of argininosuccinic aciduria.

Ashley SN, Nordin JML, Buza EL, Greig JA, Wilson JM. Ashley SN, et al. Among authors: buza el. Mol Genet Metab. 2018 Nov;125(3):241-250. doi: 10.1016/j.ymgme.2018.08.013. Epub 2018 Aug 28. Mol Genet Metab. 2018. PMID: 30253962

8

A Gene Therapy Approach to Improve Copper Metabolism and Prevent Liver Damage in a Mouse Model of Wilson Disease.

Greig JA, Nordin JML, Smith MK, Ashley SN, Draper C, Zhu Y, Bell P, Buza EL, Wilson JM. Greig JA, et al. Among authors: buza el. Hum Gene Ther Clin Dev. 2019 Mar;30(1):29-39. doi: 10.1089/humc.2018.219. Epub 2019 Feb 25. Hum Gene Ther Clin Dev. 2019. PMID: 30693797

9

Safe and Sustained Expression of Human Iduronidase After Intrathecal Administration of Adeno-Associated Virus Serotype 9 in Infant Rhesus Monkeys.

Hordeaux J, Hinderer C, Buza EL, Louboutin JP, Jahan T, Bell P, Chichester JA, Tarantal AF, Wilson JM. Hordeaux J, et al. Among authors: buza el. Hum Gene Ther. 2019 Aug;30(8):957-966. doi: 10.1089/hum.2019.012. Epub 2019 Jun 10. Hum Gene Ther. 2019. PMID: 31017018 Free PMC article.

10

Translational Feasibility of Lumbar Puncture for Intrathecal AAV Administration.

Hinderer C, Katz N, Dyer C, Goode T, Johansson J, Bell P, Richman L, Buza E, Wilson JM. Hinderer C, et al. Mol Ther Methods Clin Dev. 2020 Apr 18;17:969-974. doi: 10.1016/j.omtm.2020.04.012. eCollection 2020 Jun 12. Mol Ther Methods Clin Dev. 2020. PMID: 32420410 Free PMC article.

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