Insights into the Biology of Hearing and Deafness Revealed by Single-Cell RNA Sequencing
Ranum, Paul T., Goodwin, Alexander T., Yoshimura, Hidekane, Kolbe, Diana L., Walls, William D., Koh, Jin-Young, He, David Z.Z., Smith, Richard J.H.
Published in Cell reports (Cambridge) (12.03.2019)
Published in Cell reports (Cambridge) (12.03.2019)
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Journal Article
RNA Interference Prevents Autosomal-Dominant Hearing Loss
Shibata, Seiji B., Ranum, Paul T., Moteki, Hideaki, Pan, Bifeng, Goodwin, Alexander T., Goodman, Shawn S., Abbas, Paul J., Holt, Jeffrey R., Smith, Richard J.H.
Published in American journal of human genetics (02.06.2016)
Published in American journal of human genetics (02.06.2016)
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Journal Article
Mapping PTBP2 binding in human brain identifies SYNGAP1 as a target for therapeutic splice switching
Dawicki-McKenna, Jennine M., Felix, Alex J., Waxman, Elisa A., Cheng, Congsheng, Amado, Defne A., Ranum, Paul T., Bogush, Alexey, Dungan, Lea V., Maguire, Jean Ann, Gagne, Alyssa L., Heller, Elizabeth A., French, Deborah L., Davidson, Beverly L., Prosser, Benjamin L.
Published in Nature communications (06.05.2023)
Published in Nature communications (06.05.2023)
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Journal Article
Intravenous rAAV2/9 injection for murine cochlear gene delivery
Shibata, Seiji B., Yoshimura, Hidekane, Ranum, Paul T., Goodwin, Alexander T., Smith, Richard J. H.
Published in Scientific reports (29.08.2017)
Published in Scientific reports (29.08.2017)
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Journal Article
HOMER2, a stereociliary scaffolding protein, is essential for normal hearing in humans and mice
Azaiez, Hela, Decker, Amanda R, Booth, Kevin T, Simpson, Allen C, Shearer, A Eliot, Huygen, Patrick L M, Bu, Fengxiao, Hildebrand, Michael S, Ranum, Paul T, Shibata, Seiji B, Turner, Ann, Zhang, Yuzhou, Kimberling, William J, Cornell, Robert A, Smith, Richard J H
Published in PLoS genetics (01.03.2015)
Published in PLoS genetics (01.03.2015)
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Journal Article
Single-cell RNA-sequencing of stria vascularis cells in the adult Slc26a4 -/- mouse
Koh, Jin-Young, Affortit, Corentin, Ranum, Paul T, West, Cody, Walls, William D, Yoshimura, Hidekane, Shao, Jian Q, Mostaert, Brian, Smith, Richard J H
Published in BMC medical genomics (15.06.2023)
Published in BMC medical genomics (15.06.2023)
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Journal Article
VWA3A-derived ependyma promoter drives increased therapeutic protein secretion into the CSF
Carrell, Ellie M., Chen, Yong Hong, Ranum, Paul T., Coffin, Stephanie L., Singh, Larry N., Tecedor, Luis, Keiser, Megan S., Hudry, Eloise, Hyman, Bradley T., Davidson, Beverly L.
Published in Molecular therapy. Nucleic acids (12.09.2023)
Published in Molecular therapy. Nucleic acids (12.09.2023)
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Journal Article
Regulated control of gene therapies by drug-induced splicing
Monteys, Alex Mas, Hundley, Amiel A., Ranum, Paul T., Tecedor, Luis, Muehlmatt, Amy, Lim, Euyn, Lukashev, Dmitriy, Sivasankaran, Rajeev, Davidson, Beverly L.
Published in Nature (London) (12.08.2021)
Published in Nature (London) (12.08.2021)
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Journal Article
Comprehensive genetic testing in the clinical evaluation of 1119 patients with hearing loss
Sloan-Heggen, Christina M., Bierer, Amanda O., Shearer, A. Eliot, Kolbe, Diana L., Nishimura, Carla J., Frees, Kathy L., Ephraim, Sean S., Shibata, Seiji B., Booth, Kevin T., Campbell, Colleen A., Ranum, Paul T., Weaver, Amy E., Black-Ziegelbein, E. Ann, Wang, Donghong, Azaiez, Hela, Smith, Richard J. H.
Published in Human genetics (01.04.2016)
Published in Human genetics (01.04.2016)
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Journal Article
Toxicity after AAV delivery of RNAi expression constructs into nonhuman primate brain
Keiser, Megan S., Ranum, Paul T., Yrigollen, Carolyn M., Carrell, Ellie M., Smith, Geary R., Muehlmatt, Amy L., Chen, Yong Hong, Stein, Joel M., Wolf, Ronald L., Radaelli, Enrico, Lucas, Timothy J., Gonzalez-Alegre, Pedro, Davidson, Beverly L.
Published in Nature medicine (01.11.2021)
Published in Nature medicine (01.11.2021)
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Journal Article
Cochlear transduction via cerebrospinal fluid delivery of AAV in non-human primates
Ranum, Paul T., Tecedor, Luis, Keiser, Megan S., Chen, Yong Hong, Leib, David E., Liu, Xueyuan, Davidson, Beverly L.
Published in Molecular therapy (01.03.2023)
Published in Molecular therapy (01.03.2023)
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Journal Article
CDKL5 regulates flagellar length and localizes to the base of the flagella in Chlamydomonas
Tam, Lai-Wa, Ranum, Paul T, Lefebvre, Paul A
Published in Molecular biology of the cell (01.03.2013)
Published in Molecular biology of the cell (01.03.2013)
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Journal Article
APOE2 gene therapy reduces amyloid deposition and improves markers of neuroinflammation and neurodegeneration in a mouse model of Alzheimer disease
Jackson, Rosemary J., Keiser, Megan S., Meltzer, Jonah C., Fykstra, Dustin P., Dierksmeier, Steven E., Hajizadeh, Soroush, Kreuzer, Johannes, Morris, Robert, Melloni, Alexandra, Nakajima, Tsuneo, Tecedor, Luis, Ranum, Paul T., Carrell, Ellie, Chen, YongHong, Nishtar, Maryam A., Holtzman, David M., Haas, Wilhelm, Davidson, Beverly L., Hyman, Bradley T.
Published in Molecular therapy (01.05.2024)
Published in Molecular therapy (01.05.2024)
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Journal Article
Author Correction: Regulated control of gene therapies by drug-induced splicing
Monteys, Alex Mas, Hundley, Amiel A., Ranum, Paul T., Tecedor, Luis, Muehlmatt, Amy, Lim, Euyn, Lukashev, Dmitriy, Sivasankaran, Rajeev, Davidson, Beverly L.
Published in Nature (London) (17.02.2022)
Published in Nature (London) (17.02.2022)
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Journal Article
Immortalized striatal precursor neurons from Huntington’s disease patient-derived iPS cells as a platform for target identification and screening for experimental therapeutics
Akimov, Sergey S, Jiang, Mali, Kedaigle, Amanda J, Arbez, Nicolas, Marque, Leonard O, Eddings, Chelsy R, Ranum, Paul T, Whelan, Emma, Tang, Anthony, Wang, Ronald, DeVine, Lauren R, Talbot, Conover C, Cole, Robert N, Ratovitski, Tamara, Davidson, Beverly L, Fraenkel, Ernest, Ross, Christopher A
Published in Human molecular genetics (30.11.2021)
Published in Human molecular genetics (30.11.2021)
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Journal Article
Mutation-agnostic RNA interference with engineered replacement rescues Tmc1 -related hearing loss
Iwasa, Yoichiro, Klimara, Miles J, Yoshimura, Hidekane, Walls, William D, Omichi, Ryotaro, West, Cody A, Shibata, Seiji B, Ranum, Paul T, Smith, Richard Jh
Published in Life science alliance (01.03.2023)
Published in Life science alliance (01.03.2023)
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Journal Article
유전자 발현의 유도성 대체 스플라이싱 조절을 위한 조성물 및 방법
MONTEYS ALEJANDRO MAS, RANUM PAUL T, HUNDLEY AMIEL A, DAVIDSON BEVERLY L
Year of Publication 01.11.2022
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Year of Publication 01.11.2022
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