Intracerebroventricular Administration of AAV9-PHP.B SYN1-EmGFP Induces Widespread Transgene Expression in the Mouse and Monkey Central Nervous System von Galvan, Adriana, Petkau, Terri L., Hill, Austin M., Korecki, Andrea J., Lu, Ge, Choi, Diane, Rahman, Kazi, Simpson, Elizabeth M., Leavitt, Blair R., Smith, Yoland

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OnTarget: in silico design of MiniPromoters for targeted delivery of expression von Fornes, Oriol, Av-Shalom, Tamar V, Korecki, Andrea J, Farkas, Rachelle A, Arenillas, David J, Mathelier, Anthony, Simpson, Elizabeth M, Wasserman, Wyeth W

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Human cis-acting elements regulating escape from X-chromosome inactivation function in mouse von Peeters, Samantha B, Korecki, Andrea J, Simpson, Elizabeth M, Brown, Carolyn J

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New MiniPromoter Ple389 (ADORA2A) drives selective expression in medium spiny neurons in mice and non-human primates von de Moura Gomes, Alissandra, L. Petkau, Terri, J. Korecki, Andrea, Fornes, Oriol, Galvan, Adriana, Lu, Ge, M. Hill, Austin, Ling Lam, Siu, Yao, Anqi, A. Farkas, Rachelle, W. Wasserman, Wyeth, Smith, Yoland, M. Simpson, Elizabeth, R. Leavitt, Blair

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Intravitreal delivery of a novel AAV vector targets ON bipolar cells and restores visual function in a mouse model of complete congenital stationary night blindness von Scalabrino, Miranda L, Boye, Sanford L, Fransen, Kathryn M H, Noel, Jennifer M, Dyka, Frank M, Min, Seok Hong, Ruan, Qing, De Leeuw, Charles N, Simpson, Elizabeth M, Gregg, Ronald G, McCall, Maureen A, Peachey, Neal S, Boye, Shannon E

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Human MiniPromoters for ocular-rAAV expression in ON bipolar, cone, corneal, endothelial, Müller glial, and PAX6 cells von Korecki, Andrea J., Cueva-Vargas, Jorge L., Fornes, Oriol, Agostinone, Jessica, Farkas, Rachelle A., Hickmott, Jack W., Lam, Siu Ling, Mathelier, Anthony, Zhou, Michelle, Wasserman, Wyeth W., Di Polo, Adriana, Simpson, Elizabeth M.

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Taf1 knockout is lethal in embryonic male mice and heterozygous females show weight and movement disorders von Crombie, Elisa M, Korecki, Andrea J, Cleverley, Karen, Adair, Bethany A, Cunningham, Thomas J, Lee, Weaverly Colleen, Lengyell, Tess C, Maduro, Cheryl, Mo, Victor, Slade, Liam M, Zouhair, Ines, Fisher, Elizabeth M C, Simpson, Elizabeth M

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Human progranulin-expressing mice as a novel tool for the development of progranulin-modulating therapeutics von Petkau, Terri L., Life, Benjamin, Lu, Ge, Yang, Jasmine, Fornes, Oriol, Wasserman, Wyeth, Simpson, Elizabeth M., Leavitt, Blair R.

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Twenty-Seven Tamoxifen-Inducible iCre-Driver Mouse Strains for Eye and Brain, Including Seventeen Carrying a New Inducible-First Constitutive-Ready Allele von Korecki, Andrea J, Hickmott, Jack W, Lam, Siu Ling, Dreolini, Lisa, Mathelier, Anthony, Baker, Oliver, Kuehne, Claudia, Bonaguro, Russell J, Smith, Jillian, Tan, Chin-Vern, Zhou, Michelle, Goldowitz, Daniel, Deussing, Jan M, Stewart, A Francis, Wasserman, Wyeth W, Holt, Robert A, Simpson, Elizabeth M

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HMMR acts in the PLK1-dependent spindle positioning pathway and supports neural development von Connell, Marisa, Chen, Helen, Jiang, Jihong, Kuan, Chia-Wei, Fotovati, Abbas, Chu, Tony Lh, He, Zhengcheng, Lengyell, Tess C, Li, Huaibiao, Kroll, Torsten, Li, Amanda M, Goldowitz, Daniel, Frappart, Lucien, Ploubidou, Aspasia, Patel, Millan S, Pilarski, Linda M, Simpson, Elizabeth M, Lange, Philipp F, Allan, Douglas W, Maxwell, Christopher A

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Beyond knockouts: cre resources for conditional mutagenesis von Murray, Stephen A, Eppig, Janan T, Smedley, Damian, Simpson, Elizabeth M, Rosenthal, Nadia

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Transgene distribution and immune response after ultrasound delivery of rAAV9 and PHP.B to the brain in a mouse model of amyloidosis von Kofoed, Rikke Hahn, Heinen, Stefan, Silburt, Joseph, Dubey, Sonam, Dibia, Chinaza Lilian, Maes, Miriam, Simpson, Elizabeth M., Hynynen, Kullervo, Aubert, Isabelle

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Germline CRISPR/Cas9-Mediated Gene Editing Prevents Vision Loss in a Novel Mouse Model of Aniridia von Mirjalili Mohanna, Seyedeh Zeinab, Hickmott, Jack W., Lam, Siu Ling, Chiu, Nina Y., Lengyell, Tess C., Tam, Beatrice M., Moritz, Orson L., Simpson, Elizabeth M.

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Sonoselective delivery using ultrasound and microbubbles combined with intravenous rAAV9 CLDN5-GFP does not increase endothelial gene expression von Kofoed, Rikke Hahn, Simpson, Elizabeth M., Hynynen, Kullervo, Aubert, Isabelle

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ABE8e Corrects Pax6-Aniridic Variant in Humanized Mouse ESCs and via LNPs in Ex Vivo Cortical Neurons von Adair, Bethany A., Korecki, Andrea J., Djaksigulova, Diana, Wagner, Pamela K., Chiu, Nina Y., Lam, Siu Ling, Lengyell, Tess C., Leavitt, Blair R., Simpson, Elizabeth M.

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Expression analysis of novel striatal-enriched genes in Huntington disease von Mazarei, Gelareh, Neal, Scott J., Becanovic, Kristina, Luthi-Carter, Ruth, Simpson, Elizabeth M., Leavitt, Blair R.

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LNP-mediated delivery of CRISPR RNP for wide-spread in vivo genome editing in mouse cornea von Mirjalili Mohanna, Seyedeh Zeinab, Djaksigulova, Diana, Hill, Austin M., Wagner, Pamela K., Simpson, Elizabeth M., Leavitt, Blair R.

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rAAV-PHP.B escapes the mouse eye and causes lethality whereas rAAV9 can transduce aniridic corneal limbal stem cells without lethality von Mirjalili Mohanna, Seyedeh Zeinab, Korecki, Andrea J., Simpson, Elizabeth M.

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Frequent somatic mutations of GNAQ in uveal melanoma and blue naevi von Bastian, Boris C, Van Raamsdonk, Catherine D, Bezrookove, Vladimir, Green, Gary, Bauer, Jürgen, Gaugler, Lona, O'Brien, Joan M, Simpson, Elizabeth M, Barsh, Gregory S

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Modelling human regulatory variation in mouse: finding the function in genome-wide association studies and whole-genome sequencing von Schmouth, Jean-François, Bonaguro, Russell J, Corso-Diaz, Ximena, Simpson, Elizabeth M

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