Simpson EM - Search Results

1

Sonoselective delivery using ultrasound and microbubbles combined with intravenous rAAV9 CLDN5-GFP does not increase endothelial gene expression.

Kofoed RH, Simpson EM, Hynynen K, Aubert I. Kofoed RH, et al. Among authors: simpson em. Gene Ther. 2023 Dec;30(12):807-811. doi: 10.1038/s41434-023-00389-y. Epub 2023 Feb 13. Gene Ther. 2023. PMID: 36781945

2

New MiniPromoter Ple345 (NEFL) Drives Strong and Specific Expression in Retinal Ganglion Cells of Mouse and Primate Retina.

Simpson EM, Korecki AJ, Fornes O, McGill TJ, Cueva-Vargas JL, Agostinone J, Farkas RA, Hickmott JW, Lam SL, Mathelier A, Renner LM, Stoddard J, Zhou M, Di Polo A, Neuringer M, Wasserman WW. Simpson EM, et al. Hum Gene Ther. 2019 Mar;30(3):257-272. doi: 10.1089/hum.2018.118. Epub 2018 Oct 2. Hum Gene Ther. 2019. PMID: 30062914 Free PMC article.

3

Correction: Epistasis between Pax6^Sey and genetic background reinforces the value of defined hybrid mouse models for therapeutic trials.

Hickmott JW, Gunawardane U, Jensen K, Korecki AJ, Simpson EM. Hickmott JW, et al. Among authors: simpson em. Gene Ther. 2019 Apr;26(3-4):133. doi: 10.1038/s41434-018-0054-3. Gene Ther. 2019. PMID: 30602728 Free PMC article.

4

Human MiniPromoters for ocular-rAAV expression in ON bipolar, cone, corneal, endothelial, Müller glial, and PAX6 cells.

Korecki AJ, Cueva-Vargas JL, Fornes O, Agostinone J, Farkas RA, Hickmott JW, Lam SL, Mathelier A, Zhou M, Wasserman WW, Di Polo A, Simpson EM. Korecki AJ, et al. Among authors: simpson em. Gene Ther. 2021 Jun;28(6):351-372. doi: 10.1038/s41434-021-00227-z. Epub 2021 Feb 2. Gene Ther. 2021. PMID: 33531684 Free PMC article.

5

Intracerebroventricular Administration of AAV9-PHP.B SYN1-EmGFP Induces Widespread Transgene Expression in the Mouse and Monkey Central Nervous System.

Galvan A, Petkau TL, Hill AM, Korecki AJ, Lu G, Choi D, Rahman K, Simpson EM, Leavitt BR, Smith Y. Galvan A, et al. Among authors: simpson em. Hum Gene Ther. 2021 Jun;32(11-12):599-615. doi: 10.1089/hum.2020.301. Epub 2021 Jun 1. Hum Gene Ther. 2021. PMID: 33860682 Free PMC article.

6

Transgene distribution and immune response after ultrasound delivery of rAAV9 and PHP.B to the brain in a mouse model of amyloidosis.

Kofoed RH, Heinen S, Silburt J, Dubey S, Dibia CL, Maes M, Simpson EM, Hynynen K, Aubert I. Kofoed RH, et al. Among authors: simpson em. Mol Ther Methods Clin Dev. 2021 Oct 8;23:390-405. doi: 10.1016/j.omtm.2021.10.001. eCollection 2021 Dec 10. Mol Ther Methods Clin Dev. 2021. PMID: 34761053 Free PMC article.

7

rAAV-PHP.B escapes the mouse eye and causes lethality whereas rAAV9 can transduce aniridic corneal limbal stem cells without lethality.

Mirjalili Mohanna SZ, Korecki AJ, Simpson EM. Mirjalili Mohanna SZ, et al. Among authors: simpson em. Gene Ther. 2023 Sep;30(9):670-684. doi: 10.1038/s41434-023-00400-6. Epub 2023 Apr 19. Gene Ther. 2023. PMID: 37072572 Free PMC article.

8

PAX6 MiniPromoters drive restricted expression from rAAV in the adult mouse retina.

Hickmott JW, Chen CY, Arenillas DJ, Korecki AJ, Lam SL, Molday LL, Bonaguro RJ, Zhou M, Chou AY, Mathelier A, Boye SL, Hauswirth WW, Molday RS, Wasserman WW, Simpson EM. Hickmott JW, et al. Among authors: simpson em. Mol Ther Methods Clin Dev. 2016 Aug 10;3:16051. doi: 10.1038/mtm.2016.51. eCollection 2016. Mol Ther Methods Clin Dev. 2016. PMID: 27556059 Free PMC article.

9

Epistasis between Pax6^Sey and genetic background reinforces the value of defined hybrid mouse models for therapeutic trials.

Hickmott JW, Gunawardane U, Jensen K, Korecki AJ, Simpson EM. Hickmott JW, et al. Among authors: simpson em. Gene Ther. 2018 Dec;25(8):524-537. doi: 10.1038/s41434-018-0043-6. Epub 2018 Sep 26. Gene Ther. 2018. PMID: 30258099 Free PMC article.

10

Germline CRISPR/Cas9-Mediated Gene Editing Prevents Vision Loss in a Novel Mouse Model of Aniridia.

Mirjalili Mohanna SZ, Hickmott JW, Lam SL, Chiu NY, Lengyell TC, Tam BM, Moritz OL, Simpson EM. Mirjalili Mohanna SZ, et al. Among authors: simpson em. Mol Ther Methods Clin Dev. 2020 Mar 14;17:478-490. doi: 10.1016/j.omtm.2020.03.002. eCollection 2020 Jun 12. Mol Ther Methods Clin Dev. 2020. PMID: 32258211 Free PMC article.

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