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

Gene-based Therapy in a Mouse Model of Blue Cone Monochromacy

Authors: Yuxin Zhang, Wen-Tao Deng, Wei Du, Ping Zhu, Jie Li, Fan Xu, Jingfen Sun, Cecilia D. Gerstner, Wolfgang Baehr, Sanford L. Boye, Chen Zhao, William W. Hauswirth, Ji-jing Pang

Source: Scientific Reports, Vol 7, Iss 1, Pp 1-8 (2017)

Subject Terms: Medicine, Science

File Description: electronic resource

Relation: https://doaj.org/toc/2045-2322

Access URL: https://doaj.org/article/bc27d7c6a496437cb3861b2557b98e8d

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

Adeno-Associated Virus (AAV) Capsid Stability and Liposome Remodeling During Endo/Lysosomal pH Trafficking

Authors: Bridget Lins-Austin, Saajan Patel, Mario Mietzsch, Dewey Brooke, Antonette Bennett, Balasubramanian Venkatakrishnan, Kim Van Vliet, Adam N. Smith, Joanna R. Long, Robert McKenna, Mark Potter, Barry Byrne, Sanford L. Boye, Brian Bothner, Regine Heilbronn, Mavis Agbandje-McKenna

Source: Viruses, Vol 12, Iss 6, p 668 (2020)

Subject Terms: AAV, trafficking, capsid, liposomes, PLA2, thermostability, Microbiology, QR1-502

File Description: electronic resource

Relation: https://www.mdpi.com/1999-4915/12/6/668; https://doaj.org/toc/1999-4915

Access URL: https://doaj.org/article/c53fc790c6d747268e96cc1198cbcd18

Gene Therapy in a Large Animal Model of PDE6A-Retinitis Pigmentosa

Authors: Freya M. Mowat, Laurence M. Occelli, Joshua T. Bartoe, Kristen J. Gervais, Ashlee R. Bruewer, Janice Querubin, Astra Dinculescu, Sanford L. Boye, William W. Hauswirth, Simon M. Petersen-Jones

Source: Frontiers in Neuroscience, Vol 11 (2017)

Subject Terms: retinitis pigmentosa, Pde6 mutation, gene therapy, canine model, adeno-associated virus, Neurosciences. Biological psychiatry. Neuropsychiatry, RC321-571

File Description: electronic resource

Relation: http://journal.frontiersin.org/article/10.3389/fnins.2017.00342/full; https://doaj.org/toc/1662-453X

Access URL: https://doaj.org/article/106fda91ab70470db82c75ec58db9364

Novel Methodology for Creating Macaque Retinas with Sortable Photoreceptors and Ganglion Cells

Authors: Shreyasi Choudhury, Christianne E Strang, John J. Alexander, Miranda L. Scalabrino, Julie Lynch Hill, Daniel T. Kasuga, C. Douglas Witherspoon, Sanford L. Boye, Paul D. Gamlin, Shannon Elizabeth Boye

Source: Frontiers in Neuroscience, Vol 10 (2016)

Subject Terms: macaque, subretinal injection, retinal ganglion cells (RGCs), adeno associated virus (AAV), photoreceptors (PRs), lateral geniculate nuclei (LGN) injection, Neurosciences. Biological psychiatry. Neuropsychiatry, RC321-571

File Description: electronic resource

Relation: http://journal.frontiersin.org/Journal/10.3389/fnins.2016.00551/full; https://doaj.org/toc/1662-453X

Access URL: https://doaj.org/article/b30c8f83196a4880bb93652e93d7b597

Correction: AAV-Mediated Cone Rescue in a Naturally Occurring Mouse Model of CNGA3-Achromatopsia.

Authors: Ji-jing Pang, Wen-Tao Deng, Xufeng Dai, Bo Lei, Drew Everhart, Yumiko Umino, Jie Li, Keqing Zhang, Song Mao, Sanford L. Boye, Li Liu, Vince A. Chiodo, Xuan Liu, Wei Shi, Ye Tao, Bo Chang, William W. Hauswirth

Source: PLoS ONE, Vol 9, Iss 1 (2014)

Subject Terms: Medicine, Science

File Description: electronic resource

Relation: http://europepmc.org/articles/PMC3886851; https://doaj.org/toc/1932-6203

Access URL: https://doaj.org/article/dc2dd842f0f744fbb9fe98631e870674

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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AAV-Mediated Cone Rescue in a Naturally Occurring Mouse Model of CNGA3-Achromatopsia.

Authors: Ji-jing Pang, Wen-Tao Deng, Xufeng Dai, Bo Lei, Drew Everhart, Yumiko Umino, Jie Li, Keqing Zhang, Song Mao, Sanford L. Boye, Li Liu, Chiodo, Vince A., Xuan Liu, Wei Shi, Ye Tao, Bo Chang5, Hauswirth, William W.

Source: PLoS ONE; Apr2012, Vol. 7 Issue 4, p1-9, 9p

Subject Terms: THERAPEUTICS, VISION disorders, BLINDNESS, GENE therapy, VISUAL acuity, CYCLIC nucleotides, CONTRAST sensitivity (Vision)

Human RPE65Gene Therapy for Leber Congenital Amaurosis: Persistence of Early Visual Improvements and Safety at 1 Year.

Authors: Artur V. Cideciyan, William W. Hauswirth, Tomas S. Aleman, Shalesh Kaushal, Sharon B. Schwartz, Sanford L. Boye, Elizabeth A.M. Windsor, Thomas J. Conlon, Alexander Sumaroka, Ji-jing Pang, Alejandro J. Roman, Barry J. Byrne, Samuel G. Jacobson

Source: Human Gene Therapy; Aug2009, Vol. 20 Issue 9, p999-1004, 6p

Treatment of Leber Congenital Amaurosis Due to RPE65Mutations by Ocular Subretinal Injection of Adeno-Associated Virus Gene Vector Short-Term Results of a Phase I Trial.

Authors: William W. Hauswirth, Tomas S. Aleman, Shalesh Kaushal, Artur V. Cideciyan, Sharon B. Schwartz, Lili Wang, Thomas J. Conlon, Sanford L. Boye, Terence R. Flotte, Barry J. Byrne, Samuel G. Jacobson

Source: Human Gene Therapy; Oct2008, Vol. 19 Issue 10, p979-990, 12p

Safety in Nonhuman Primates of Ocular AAV2-RPE65, a Candidate Treatment for Blindness in Leber Congenital Amaurosis.

Authors: Samuel G. Jacobson, Sanford L. Boye, Tomas S. Aleman, Thomas J. Conlon, Caroline J. Zeiss, Alejandro J. Roman, Artur V. Cideciyan, Sharon B. Schwartz, Andras M. Komaromy, Michelle Doobrajh, Andy Y. Cheung, Alexander Sumaroka, Susan E. Pearce-Kelling, Gustavo D. Aguirre, Shalesh Kaushal, Albert M. Maguire, Terence R. Flotte, William W. Hauswirth

Source: Human Gene Therapy; Aug2006, Vol. 17 Issue 8, p845-858, 14p