TUBB4B is essential for the cytoskeletal architecture of cochlear supporting cells and motile cilia development

Authors: Urikhan Sanzhaeva, Helen Boyd-Pratt, Philip T. R. Bender, Thamaraiselvi Saravanan, Scott B. Rhodes, Tongju Guan, Neil Billington, Shannon E. Boye, Christopher L. Cunningham, Charles T. Anderson, Visvanathan Ramamurthy

Source: Communications Biology, Vol 7, Iss 1, Pp 1-18 (2024)

Subject Terms: Biology (General), QH301-705.5

File Description: electronic resource

Relation: https://doaj.org/toc/2399-3642

Access URL: https://doaj.org/article/2bce1a002054404696b0451fc39c920b

Safety and improved efficacy signals following gene therapy in childhood blindness caused by GUCY2D mutations

Authors: Samuel G. Jacobson, Artur V. Cideciyan, Allen C. Ho, Igor V. Peshenko, Alexandra V. Garafalo, Alejandro J. Roman, Alexander Sumaroka, Vivian Wu, Arun K. Krishnan, Rebecca Sheplock, Sanford L. Boye, Alexander M. Dizhoor, Shannon E. Boye

Source: iScience, Vol 27, Iss 11, Pp 111048- (2024)

Subject Terms: Science

File Description: electronic resource

Relation: http://www.sciencedirect.com/science/article/pii/S2589004224022739; https://doaj.org/toc/2589-0042

Access URL: https://doaj.org/article/d871dc2a0b694da9942e276b248c9408

Development of an AAV-CRISPR-Cas9-based treatment for dominant cone-rod dystrophy 6

Authors: Russell W. Mellen, Kaitlyn R. Calabro, K. Tyler McCullough, Sean M. Crosson, Alejandro de la Cova, Diego Fajardo, Emily Xu, Sanford L. Boye, Shannon E. Boye

Source: Molecular Therapy: Methods & Clinical Development, Vol 30, Iss , Pp 48-64 (2023)

Subject Terms: AAV, adeno-associated virus, GUCY2D, cone-rod dystrophy, CORD6, gene therapy, Genetics, QH426-470, Cytology, QH573-671

File Description: electronic resource

Relation: http://www.sciencedirect.com/science/article/pii/S2329050123000827; https://doaj.org/toc/2329-0501

Access URL: https://doaj.org/article/e73f2be0ddd74708a9903c786112e07c

Dual-AAV vector-mediated expression of MYO7A improves vestibular function in a mouse model of Usher syndrome 1B

Authors: Samantha C. Lau, Mhamed Grati, Kevin Isgrig, Moaz Sinan, Kaitlyn R. Calabro, Jianliang Zhu, Yasuko Ishibashi, Zeynep Ozgur, Talah Wafa, Inna A. Belyantseva, Tracy Fitzgerald, Thomas B. Friedman, Sanford L. Boye, Shannon E. Boye, Wade W. Chien

Source: Molecular Therapy: Methods & Clinical Development, Vol 30, Iss , Pp 534-545 (2023)

Subject Terms: gene therapy, hearing loss, Usher syndrome, USH1B, MYO7A, AAV, Genetics, QH426-470, Cytology, QH573-671

File Description: electronic resource

Relation: http://www.sciencedirect.com/science/article/pii/S2329050123001262; https://doaj.org/toc/2329-0501

Access URL: https://doaj.org/article/ea475ade936f41d5b9fc0d539fc7be4e

Preclinical studies in support of phase I/II clinical trials to treat GUCY2D-associated Leber congenital amaurosis

Authors: Sanford L. Boye, Catherine O’Riordan, James Morris, Michael Lukason, David Compton, Rena Baek, Dana M. Elmore, James.J. Peterson, Diego Fajardo, K. Tyler McCullough, Abraham Scaria, Alison McVie-Wylie, Shannon E. Boye

Source: Molecular Therapy: Methods & Clinical Development, Vol 28, Iss , Pp 129-145 (2023)

Subject Terms: AAV, adeno-associated virus, GUCY2D, Leber congenital amaurosis, LCA, LCA1, Genetics, QH426-470, Cytology, QH573-671

File Description: electronic resource

Relation: http://www.sciencedirect.com/science/article/pii/S2329050122001802; https://doaj.org/toc/2329-0501

Access URL: https://doaj.org/article/a3ce88c96c0141e087c17132b69d17e5

Night vision restored in days after decades of congenital blindness

Authors: Samuel G. Jacobson, Artur V. Cideciyan, Allen C. Ho, Alejandro J. Roman, Vivian Wu, Alexandra V. Garafalo, Alexander Sumaroka, Arun K. Krishnan, Malgorzata Swider, Abraham A. Mascio, Christine N. Kay, Dan Yoon, Kenji P. Fujita, Sanford L. Boye, Igor V. Peshenko, Alexander M. Dizhoor, Shannon E. Boye

Source: iScience, Vol 25, Iss 10, Pp 105274- (2022)

Subject Terms: Clinical genetics, Health sciences, Medicine, Science

File Description: electronic resource

Relation: http://www.sciencedirect.com/science/article/pii/S2589004222015462; https://doaj.org/toc/2589-0042

Access URL: https://doaj.org/article/00767dff06174533bc77d98fb800d731

Utilizing minimally purified secreted rAAV for rapid and cost-effective manipulation of gene expression in the CNS

Authors: Marshall S. Goodwin, Cara L. Croft, Hunter S. Futch, Daniel Ryu, Carolina Ceballos-Diaz, Xuefei Liu, Giavanna Paterno, Catalina Mejia, Doris Deng, Kimberly Menezes, Laura Londono, Kefren Arjona, Mary Parianos, Van Truong, Eva Rostonics, Amanda Hernandez, Sanford L. Boye, Shannon E. Boye, Yona Levites, Pedro E. Cruz, Todd E. Golde

Source: Molecular Neurodegeneration, Vol 15, Iss 1, Pp 1-16 (2020)

Subject Terms: Adeno-associated virus, Capsid pseudo-type, AAV production, Gene delivery, Central nervous system, Neurology. Diseases of the nervous system, RC346-429, Geriatrics, RC952-954.6

File Description: electronic resource

Relation: http://link.springer.com/article/10.1186/s13024-020-00361-z; https://doaj.org/toc/1750-1326

Access URL: https://doaj.org/article/2a178227546947689b7deca1eadc1b03

Safety and improved efficacy signals following gene therapy in childhood blindness caused by GUCY2D mutations

Authors: Samuel G. Jacobson, Artur V. Cideciyan, Allen C. Ho, Igor V. Peshenko, Alexandra V. Garafalo, Alejandro J. Roman, Alexander Sumaroka, Vivian Wu, Arun K. Krishnan, Rebecca Sheplock, Sanford L. Boye, Alexander M. Dizhoor, Shannon E. Boye

Source: iScience, Vol 24, Iss 5, Pp 102409- (2021)

Subject Terms: Clinical Genetics, Clinical Finding, Techniques in Genetics, Science

File Description: electronic resource

Relation: http://www.sciencedirect.com/science/article/pii/S2589004221003771; https://doaj.org/toc/2589-0042

Access URL: https://doaj.org/article/42b6c97445f342b5b2678c7eb8a835d4

Rationally Engineered AAV Capsids Improve Transduction and Volumetric Spread in the CNS

Authors: Nicholas M. Kanaan, Rhyomi C. Sellnow, Sanford L. Boye, Ben Coberly, Antonette Bennett, Mavis Agbandje-McKenna, Caryl E. Sortwell, William W. Hauswirth, Shannon E. Boye, Fredric P. Manfredsson

Source: Molecular Therapy: Nucleic Acids, Vol 8, Iss , Pp 184-197 (2017)

Subject Terms: Therapeutics. Pharmacology, RM1-950

File Description: electronic resource

Relation: http://www.sciencedirect.com/science/article/pii/S216225311730197X; https://doaj.org/toc/2162-2531

Access URL: https://doaj.org/article/6d0d371fda0549eea1c15d7cb44b1c23

A Novel Mouse Model of MYO7A USH1B Reveals Auditory and Visual System Haploinsufficiencies

Authors: Kaitlyn R. Calabro, Sanford L. Boye, Shreyasi Choudhury, Diego Fajardo, James J. Peterson, Wei Li, Sean M. Crosson, Mi-Jung Kim, Dalian Ding, Richard Salvi, Shinichi Someya, Shannon E. Boye

Source: Frontiers in Neuroscience, Vol 13 (2019)

Subject Terms: retina, cochlea, Usher syndrome, myosin-7A, vision, hearing, Neurosciences. Biological psychiatry. Neuropsychiatry, RC321-571

File Description: electronic resource

Relation: https://www.frontiersin.org/article/10.3389/fnins.2019.01255/full; https://doaj.org/toc/1662-453X

Access URL: https://doaj.org/article/8c3c907278374492bd371de6e22e0688

Correction: Targeting Photoreceptors via Intravitreal Delivery Using Novel, Capsid-Mutated AAV Vectors.

Authors: Christine N. Kay, Renee C. Ryals, George V. Aslanidi, Seok Hong Min, Qing Ruan, Jingfen Sun, Frank M. Dyka, Daniel Kasuga, Andrea E. Ayala, Kim Van Vliet, Mavis Agbandje-McKenna, William W. Hauswirth, Sanford L. Boye, Shannon E. Boye

Source: PLoS ONE, Vol 8, Iss 9 (2013)

Subject Terms: Medicine, Science

File Description: electronic resource

Relation: http://europepmc.org/articles/PMC3783628; https://doaj.org/toc/1932-6203

Access URL: https://doaj.org/article/1e7d397ee6504d4991261a14ef4ad389

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Gene delivery to mitochondria by targeting modified adenoassociated virus suppresses Leber's hereditary optic neuropathy in a mouse model.

Authors: Hong Yu, Koilkonda, Rajeshwari D., Tsung-Han Chou, Porciatti, Vittorio, Ozdemir, Sacide S., Chiodo, Vince, Boye, Sanford L., Shannon E. Boye, Hauswirth, William W., Lewin, Alfred S., Guy, John

Source: Proceedings of the National Academy of Sciences of the United States of America; 5/15/2012, Vol. 109 Issue 20, p7599-7599, 1p

Subject Terms: LEBER'S hereditary optic atrophy, ANIMAL models in research

Improving retinal vascular endothelial cell tropism through rational rAAV capsid design.

Authors: Ramesh Periasamy, Dwani D Patel, Sanford L Boye, Shannon E Boye, Daniel M Lipinski

Source: PLoS ONE, Vol 18, Iss 5, p e0285370 (2023)

Subject Terms: Medicine, Science

File Description: electronic resource

Relation: https://journals.plos.org/plosone/article/file?id=10.1371/journal.pone.0285370&type=printable; https://doaj.org/toc/1932-6203

Access URL: https://doaj.org/article/414639d9a1454b6e8f76ccf60d3e82e0

SARM1 depletion rescues NMNAT1-dependent photoreceptor cell death and retinal degeneration

Authors: Yo Sasaki, Hiroki Kakita, Shunsuke Kubota, Abdoulaye Sene, Tae Jun Lee, Norimitsu Ban, Zhenyu Dong, Joseph B Lin, Sanford L Boye, Aaron DiAntonio, Shannon E Boye, Rajendra S Apte, Jeffrey Milbrandt

Source: eLife, Vol 9 (2020)

Subject Terms: retinal degenerations, NAD+, SARM1, NMNAT1, LCA9, axonal degeneration, Medicine, Science, Biology (General), QH301-705.5

File Description: electronic resource

Relation: https://elifesciences.org/articles/62027; https://doaj.org/toc/2050-084X

Access URL: https://doaj.org/article/4275e0ca179049eabb78a4c6222a8885

Capsid Mutated Adeno-Associated Virus Delivered to the Anterior Chamber Results in Efficient Transduction of Trabecular Meshwork in Mouse and Rat.

Authors: Barbara Bogner, Sanford L Boye, Seok Hong Min, James J Peterson, Qing Ruan, Zhonghong Zhang, Herbert A Reitsamer, William W Hauswirth, Shannon E Boye

Source: PLoS ONE, Vol 10, Iss 6, p e0128759 (2015)

Subject Terms: Medicine, Science

File Description: electronic resource

Relation: http://europepmc.org/articles/PMC4460001?pdf=render; https://doaj.org/toc/1932-6203

Access URL: https://doaj.org/article/8834ec7065cb423ca04394031b961bd8

Natural history of cone disease in the murine model of Leber congenital amaurosis due to CEP290 mutation: determining the timing and expectation of therapy.

Authors: Shannon E Boye, Wei-Chieh Huang, Alejandro J Roman, Alexander Sumaroka, Sanford L Boye, Renee C Ryals, Melani B Olivares, Qing Ruan, Budd A Tucker, Edwin M Stone, Anand Swaroop, Artur V Cideciyan, William W Hauswirth, Samuel G Jacobson

Source: PLoS ONE, Vol 9, Iss 3, p e92928 (2014)

Subject Terms: Medicine, Science

File Description: electronic resource

Relation: http://europepmc.org/articles/PMC3966841?pdf=render; https://doaj.org/toc/1932-6203

Access URL: https://doaj.org/article/9bceffafe04b4e8ba21a5c75da5683f4

Targeted CNS delivery using human MiniPromoters and demonstrated compatibility with adeno-associated viral vectors

Authors: Charles N de Leeuw, Frank M Dyka, Sanford L Boye, Stéphanie Laprise, Michelle Zhou, Alice Y Chou, Lisa Borretta, Simone C McInerny, Kathleen G Banks, Elodie Portales-Casamar, Magdalena I Swanson, Cletus A D'Souza, Shannon E Boye, Steven JM Jones, Robert A Holt, Daniel Goldowitz, William W Hauswirth, Wyeth W Wasserman, Elizabeth M Simpson

Source: Molecular Therapy: Methods & Clinical Development, Vol 1, Iss C (2014)

Subject Terms: Genetics, QH426-470, Cytology, QH573-671

File Description: electronic resource

Relation: http://www.sciencedirect.com/science/article/pii/S2329050116300705; https://doaj.org/toc/2329-0501

Access URL: https://doaj.org/article/a4992868fae84ac68f19247787a4fd6f

Targeting photoreceptors via intravitreal delivery using novel, capsid-mutated AAV vectors.

Authors: Christine N Kay, Renee C Ryals, George V Aslanidi, Seok Hong Min, Qing Ruan, Jingfen Sun, Frank M Dyka, Daniel Kasuga, Andrea E Ayala, Kim Van Vliet, Mavis Agbandje-McKenna, William W Hauswirth, Sanford L Boye, Shannon E Boye

Source: PLoS ONE, Vol 8, Iss 4, p e62097 (2013)

Subject Terms: Medicine, Science

File Description: electronic resource

Relation: http://europepmc.org/articles/PMC3637363?pdf=render; https://doaj.org/toc/1932-6203

Access URL: https://doaj.org/article/acb82117f6a642269cce77147bb4a790