Frair EC - Search Results

1

CRISPR-Cas9 homology-independent targeted integration of exons 1-19 restores full-length dystrophin in mice.

Stephenson AA, Nicolau S, Vetter TA, Dufresne GP, Frair EC, Sarff JE, Wheeler GL, Kelly BJ, White P, Flanigan KM. Stephenson AA, et al. Among authors: frair ec. Mol Ther Methods Clin Dev. 2023 Aug 18;30:486-499. doi: 10.1016/j.omtm.2023.08.009. eCollection 2023 Sep 14. Mol Ther Methods Clin Dev. 2023. PMID: 37706184 Free PMC article.

2

Lack of Toxicity in Nonhuman Primates Receiving Clinically Relevant Doses of an AAV9.U7snRNA Vector Designed to Induce DMD Exon 2 Skipping.

Gushchina LV, Frair EC, Rohan N, Bradley AJ, Simmons TR, Chavan HD, Chou HJ, Eggers M, Waldrop MA, Wein N, Flanigan KM. Gushchina LV, et al. Among authors: frair ec. Hum Gene Ther. 2021 Sep;32(17-18):882-894. doi: 10.1089/hum.2020.286. Epub 2021 May 7. Hum Gene Ther. 2021. PMID: 33406986 Free PMC article.

3

Direct Reprogramming of Human Fibroblasts into Myoblasts to Investigate Therapies for Neuromuscular Disorders.

Almeida CF, Frair EC, Huang N, Neinast R, McBride KL, Weiss RB, Flanigan KM, Wein N. Almeida CF, et al. Among authors: frair ec. J Vis Exp. 2021 Apr 3;(170). doi: 10.3791/61991. J Vis Exp. 2021. PMID: 33871464

4

Absence of Significant Off-Target Splicing Variation with a U7snRNA Vector Targeting DMD Exon 2 Duplications.

Wein N, Dunn DM, Waldrop MA, Gushchina LV, Frair EC, Weiss RB, Flanigan KM. Wein N, et al. Among authors: frair ec. Hum Gene Ther. 2021 Nov;32(21-22):1346-1359. doi: 10.1089/hum.2020.315. Epub 2021 Jul 20. Hum Gene Ther. 2021. PMID: 34060935

5

Automated immunofluorescence analysis for sensitive and precise dystrophin quantification in muscle biopsies.

Vetter TA, Nicolau S, Bradley AJ, Frair EC, Flanigan KM. Vetter TA, et al. Among authors: frair ec. Neuropathol Appl Neurobiol. 2022 Apr;48(3):e12785. doi: 10.1111/nan.12785. Epub 2021 Dec 28. Neuropathol Appl Neurobiol. 2022. PMID: 34847621 Free PMC article.

6

Systemic delivery of an AAV9 exon-skipping vector significantly improves or prevents features of Duchenne muscular dystrophy in the Dup2 mouse.

Wein N, Vetter TA, Vulin A, Simmons TR, Frair EC, Bradley AJ, Gushchina LV, Almeida CF, Huang N, Lesman D, Rajakumar D, Weiss RB, Flanigan KM. Wein N, et al. Among authors: frair ec. Mol Ther Methods Clin Dev. 2022 Jul 11;26:279-293. doi: 10.1016/j.omtm.2022.07.005. eCollection 2022 Sep 8. Mol Ther Methods Clin Dev. 2022. PMID: 35949298 Free PMC article.

7

A first-in-human phase I/IIa gene transfer clinical trial for Duchenne muscular dystrophy using rAAVrh74.MCK.GALGT2.

Flanigan KM, Vetter TA, Simmons TR, Iammarino M, Frair EC, Rinaldi F, Chicoine LG, Harris J, Cheatham JP, Cheatham SL, Boe B, Waldrop MA, Zygmunt DA, Packer D, Martin PT. Flanigan KM, et al. Among authors: frair ec. Mol Ther Methods Clin Dev. 2022 Sep 2;27:47-60. doi: 10.1016/j.omtm.2022.08.009. eCollection 2022 Dec 8. Mol Ther Methods Clin Dev. 2022. PMID: 36186954 Free PMC article.

8

Systemic PPMO-mediated dystrophin expression in the Dup2 mouse model of Duchenne muscular dystrophy.

Gushchina LV, Vetter TA, Frair EC, Bradley AJ, Grounds KM, Lay JW, Huang N, Suhaiba A, Schnell FJ, Hanson G, Simmons TR, Wein N, Flanigan KM. Gushchina LV, et al. Among authors: frair ec. Mol Ther Nucleic Acids. 2022 Nov 9;30:479-492. doi: 10.1016/j.omtn.2022.10.025. eCollection 2022 Dec 13. Mol Ther Nucleic Acids. 2022. PMID: 36420217 Free PMC article.

9

Persistence of exon 2 skipping and dystrophin expression at 18 months after U7snRNA-mediated therapy in the Dup2 mouse model.

Gushchina LV, Bradley AJ, Vetter TA, Lay JW, Rohan NL, Frair EC, Wein N, Flanigan KM. Gushchina LV, et al. Among authors: frair ec. Mol Ther Methods Clin Dev. 2023 Oct 26;31:101144. doi: 10.1016/j.omtm.2023.101144. eCollection 2023 Dec 14. Mol Ther Methods Clin Dev. 2023. PMID: 38027058 Free PMC article.

10

PRMT5-PTEN molecular pathway regulates senescence and self-renewal of primary glioblastoma neurosphere cells.

Banasavadi-Siddegowda YK, Russell L, Frair E, Karkhanis VA, Relation T, Yoo JY, Zhang J, Sif S, Imitola J, Baiocchi R, Kaur B. Banasavadi-Siddegowda YK, et al. Oncogene. 2017 Jan 12;36(2):263-274. doi: 10.1038/onc.2016.199. Epub 2016 Jun 13. Oncogene. 2017. PMID: 27292259 Free PMC article.

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