Zobrazeno 1 - 10
of 37
pro vyhledávání: '"Sundeep Chandra"'
Autor:
Jason Pinkstaff, Emma McCullagh, Anita Grover, Andrew C. Melton, Anu Cherukuri, Jill CM Wait, Annalisa Nguyen, Mark T. Butt, Jami L. Trombley, Randall P. Reed, Eric.L. Adams, Robert B. Boyd, Sundeep Chandra, Joshua Henshaw, Charles A. O’Neill, Eric Zanelli, Joseph Kovalchin
Publikováno v:
Toxicology Reports, Vol 10, Iss , Pp 357-366 (2023)
Mucopolysaccharidosis Type IIIB (MPS IIIB) is an ultrarare, fatal pediatric disease with no approved therapy. It is caused by mutations in the gene encoding for lysosomal enzyme alpha-N-acetylglucosaminidase (NAGLU). Tralesinidase alfa (TA) is a fusi
Externí odkaz:
https://doaj.org/article/cfbd12d153c245b5adea0f11f64be6c8
Autor:
Lening Zhang, Bridget Yates, Ryan Murphy, Su Liu, Lin Xie, Britta Handyside, Choong-Ryoul Sihn, Taren Bouwman, Nicole Galicia, Danielle Tan, Carlos Fonck, Jeremy Arens, Annie Clark, Weiming Zhang, Sundeep Chandra, Jaydeep Srimani, Jennifer Holcomb, Andrea Van Tuyl, Joshua Henshaw, Christian Vettermann, Silvia Siso, Cheng Su, Sherry Bullens, Stuart Bunting, Charles O’Neill, Sylvia Fong
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 26, Iss , Pp 519-531 (2022)
Valoctocogene roxaparvovec (AAV5-hFVIII-SQ) gene transfer provided reduced bleeding for adult clinical trial participants with severe hemophilia A. However, pediatric outcomes are unknown. Using a mouse model of hemophilia A, we investigated the effe
Externí odkaz:
https://doaj.org/article/19b2dc22c74b4524a6290d6684a6c08b
Autor:
Michael C. Babcock, Christina R. Mikulka, Bing Wang, Sanjay Chandriani, Sundeep Chandra, Yue Xu, Katherine Webster, Ying Feng, Hemanth R. Nelvagal, Alex Giaramita, Bryan K. Yip, Melanie Lo, Xuntian Jiang, Qi Chao, Josh C. Woloszynek, Yuqiao Shen, Shripad Bhagwat, Mark S. Sands, Brett E. Crawford
Publikováno v:
Scientific Reports, Vol 11, Iss 1, Pp 1-17 (2021)
Abstract Krabbe disease (KD) and metachromatic leukodystrophy (MLD) are caused by accumulation of the glycolipids galactosylceramide (GalCer) and sulfatide and their toxic metabolites psychosine and lysosulfatide, respectively. We discovered a potent
Externí odkaz:
https://doaj.org/article/b4baa35aa3c440d78cac8c75d11ceb69
Autor:
Toloo Taghian, Jillian Gallagher, Erin Batcho, Caitlin Pullan, Tim Kuchel, Thomas Denney, Raj Perumal, Shamika Moore, Robb Muirhead, Paul Herde, Daniel Johns, Chris Christou, Amanda Taylor, Thomas Passler, Sanjana Pulaparthi, Erin Hall, Sundeep Chandra, Charles A. O’Neill, Heather Gray-Edwards
Publikováno v:
Journal of Huntington's disease. 11(4)
Background: Huntington’s disease (HD) is a fatal neurodegenerative autosomal dominant disorder with prevalence of 1 : 20000 that has no effective treatment to date. Translatability of candidate therapeutics could be enhanced by additional testing i
Autor:
Joanna Barton, Naoki Yoshimura, Takahiro Shimizu, Katsumi Kadekawa, Naoki Kawamorita, Sundeep Chandra, Alan J. Russell, Philip Stewart Turnbull, Masahiro Kurobe, Takahito Kambara
Publikováno v:
BJU International. 125:911-919
Objectives To report the effect of a selective androgen receptor modulators (SARMs) on the urethral continence mechanisms in a rat model of stress urinary incontinence (SUI) induced by bilateral ovariectomy (OVX). Materials and methods Female Sprague
Autor:
Katherine A. Webster, Bryan K. Yip, Yue Xu, Christina R. Mikulka, Alex Giaramita, Brett E. Crawford, Michael C. Babcock, Hemanth R. Nelvagal, Sanjay Chandriani, Xuntian Jiang, Ying Feng, Melanie Lo, Sundeep Chandra, Josh C. Woloszynek, Shripad Bhagwat, Qi Chao, Yuqiao Shen, Mark S. Sands, Bing Wang
Publikováno v:
Scientific Reports
Scientific Reports, Vol 11, Iss 1, Pp 1-17 (2021)
Scientific Reports, Vol 11, Iss 1, Pp 1-17 (2021)
Krabbe disease (KD) and metachromatic leukodystrophy (MLD) are caused by accumulation of the glycolipids galactosylceramide (GalCer) and sulfatide and their toxic metabolites psychosine and lysosulfatide, respectively. We discovered a potent and sele
Autor:
Carlos Fonck, Cheng Su, Jeremy Arens, Elli Koziol, Jaydeep Srimani, Joshua Henshaw, Andrea Van Tuyl, Sundeep Chandra, Christian Vettermann, Charles A. O’Neill
Publikováno v:
Gene therapy.
Valoctocogene roxaparvovec (AAV5-hFVIII-SQ) is an adeno-associated virus serotype five gene therapy under investigation for the treatment of hemophilia A. Herein, we assessed the potential for germline transmission of AAV5-hFVIII-SQ in mice. Male B6.
Publikováno v:
Toxicologic Pathology. 47:656-660
A therapeutic option for monogenic disorders is gene therapy with ex vivo-transduced autologous hematopoietic stem cells (HSCs). Safety or efficacy studies of ex vivo-modified HSCs are conducted in humanized mouse models after ablation of the murine
Autor:
Jey W. Koehler, Ashley N. Randle, Jillian Gallagher, Amanda L. Gross, Elise B. Diffie, Brett D. Story, Sundeep Chandra, Sara Carl, Kayly Nielsen, Paul Cuddon, Miguel Sena-Esteves, Edwin H. Kolodny, Deborah Fernau, Amanda R. Taylor, Douglas R. Martin, Carly Corado, Xuntian Jiang, Heather L. Gray-Edwards, Annie S. Maguire, Toloo Taghian, Siauna Johnson
Publikováno v:
Mol Genet Metab
Tay-Sachs disease (TSD) is a fatal neurodegenerative disease caused by a deficiency of the enzyme β-N-acetylhexosaminidase A (HexA). TSD naturally occurs in Jacob sheep is the only experimental model of TSD. TSD in sheep recapitulates neurologic fea
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::69a0c7cda10ecdd0ead870203e1fd7ba
https://europepmc.org/articles/PMC8811770/
https://europepmc.org/articles/PMC8811770/
Autor:
Sundeep Chandra, Katherine Bibi, Charles A. O'Neill, John Sinclair, Daniella P. Vansteenkiste, Martin L. Katz, Jacqueline W. Pearce, Annalisa Nguyen, Grace Robinson Kick, Stefanie Lim, Leilani J. Castaner, Rebecca E.H. Whiting
Publikováno v:
Exp Eye Res
CLN2 neuronal ceroid lipofuscinosis is a hereditary neurodegenerative disorder characterized by progressive vision loss, neurological decline, and seizures. CLN2 disease results from mutations in TPP1 that encodes the lysosomal enzyme tripeptidyl pep
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::f993cf47b7dc132a777799fb9a5ae6ab
https://europepmc.org/articles/PMC7484259/
https://europepmc.org/articles/PMC7484259/