Zobrazeno 1 - 10
of 20
pro vyhledávání: '"Diane Golebiowski"'
Autor:
Laura P. van Lieshout, Miranda Rubin, Katrina Costa-Grant, Stacy Ota, Diane Golebiowski, Troy Panico, Eli Wiberg, Klaudia Szymczak, Richard Gilmore, Marissa Stanvick, Brenda Burnham, Jeff Gagnon, Ifeyinwa Iwuchukwu, Guang Yang, Iraj Ghazi, Alex Meola, Ryan Dickerson, Thomas Thiers, Luke Mustich, April Hayes, Israel Rivas, Jason Lotterhand, Nancy Avila, James McGivney, Jin Yin, Tim Kelly
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 29, Iss , Pp 426-436 (2023)
Transient transfection of mammalian cells using plasmid DNA is a standard method to produce adeno-associated virus (AAV) vectors allowing for flexible and scalable manufacture. Typically, three plasmids are used to encode the necessary components to
Externí odkaz:
https://doaj.org/article/25fd1b8281394ec9b15853de27025cb5
Autor:
Meenakshi Sundaram Kumar, Megan E. Fowler-Magaw, Daniel Kulick, Sivakumar Boopathy, Del Hayden Gadd, Melissa Rotunno, Catherine Douthwright, Diane Golebiowski, Issa Yusuf, Zuoshang Xu, Robert H. Brown, Miguel Sena-Esteves, Alison L. O'Neil, Daryl A. Bosco
Publikováno v:
International Journal of Molecular Sciences, Vol 23, Iss 24, p 16013 (2022)
ALS-linked mutations induce aberrant conformations within the SOD1 protein that are thought to underlie the pathogenic mechanism of SOD1-mediated ALS. Although clinical trials are underway for gene silencing of SOD1, these approaches reduce both wild
Externí odkaz:
https://doaj.org/article/1f504476dde54f76be4c1437cd4cd3b1
Autor:
Tahnee L. Kennedy, Simon Guiraud, Ben Edwards, Sarah Squire, Lee Moir, Arran Babbs, Guy Odom, Diane Golebiowski, Joel Schneider, Jeffrey S. Chamberlain, Kay E. Davies
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 11, Iss , Pp 92-105 (2018)
Duchenne muscular dystrophy (DMD) is an X-linked muscle-wasting disease caused by mutations in the dystrophin gene. DMD boys are wheelchair-bound around 12 years and generally survive into their twenties. There is currently no effective treatment exc
Externí odkaz:
https://doaj.org/article/b4cdbb1c898240c9ae805bf8b86d8caa
Autor:
Tatianna Wai Ying Wong, Abdalla Ahmed, Grace Yang, Eleonora Maino, Sydney Steiman, Elzbieta Hyatt, Parry Chan, Kyle Lindsay, Nicole Wong, Diane Golebiowski, Joel Schneider, Paul Delgado-Olguín, Evgueni A. Ivakine, Ronald D. Cohn
Publikováno v:
Disease Models & Mechanisms, Vol 13, Iss 9 (2020)
Duchenne muscular dystrophy (DMD) is a life-threatening neuromuscular disease caused by the lack of dystrophin, resulting in progressive muscle wasting and locomotor dysfunctions. By adulthood, almost all patients also develop cardiomyopathy, which i
Externí odkaz:
https://doaj.org/article/331a1839953f44fc84ba83fead6d8655
Autor:
Maria L. Allende, Emily K. Cook, Bridget C. Larman, Adrienne Nugent, Jacqueline M. Brady, Diane Golebiowski, Miguel Sena-Esteves, Cynthia J. Tifft, Richard L. Proia
Publikováno v:
Journal of Lipid Research, Vol 59, Iss 3, Pp 550-563 (2018)
Sandhoff disease, one of the GM2 gangliosidoses, is a lysosomal storage disorder characterized by the absence of β-hexosaminidase A and B activity and the concomitant lysosomal accumulation of its substrate, GM2 ganglioside. It features catastrophic
Externí odkaz:
https://doaj.org/article/92b131018d354437940c5128314b85f9
Autor:
Anastasia Khvorova, Miguel Sena-Esteves, Jonathan K. Watts, Michael P. Moazami, Dimas Echeverria, Diane Golebiowski, Andrew H. Coles, Maire F. Osborn
Glioblastoma (GBM) is the most common and lethal form of primary brain tumor with dismal median and 2-year survivals of 14.5 months and 18%, respectively. The paucity of new therapeutic agents stems from the complex biology of a highly adaptable tumo
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::01d609951d687c95196190cb7b9e136c
https://doi.org/10.1158/1535-7163.c.6538093.v1
https://doi.org/10.1158/1535-7163.c.6538093.v1
Autor:
Anastasia Khvorova, Miguel Sena-Esteves, Jonathan K. Watts, Michael P. Moazami, Dimas Echeverria, Diane Golebiowski, Andrew H. Coles, Maire F. Osborn
Figure S1. Cytotoxicity of Chol-hsiRNAs in vitro Table S1. Tumor-associated luciferase activity Table S2. Chol-hsiRNA sequences and chemical modification patterns
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::0f9b0bae80aadbb09e57195d5697103d
https://doi.org/10.1158/1535-7163.22505506.v1
https://doi.org/10.1158/1535-7163.22505506.v1
Autor:
Sharla M. Birch, Michael W. Lawlor, Thomas J. Conlon, Lee-Jae Guo, Julie M. Crudele, Eleanor C. Hawkins, Peter P. Nghiem, Mihye Ahn, Hui Meng, Margaret J. Beatka, Brittany A. Fickau, Juan C. Prieto, Martin A. Styner, Michael J. Struharik, Courtney Shanks, Kristy J. Brown, Diane Golebiowski, Amanda K. Bettis, Cynthia J. Balog-Alvarez, Nathalie Clement, Kirsten E. Coleman, Manuela Corti, Xiufang Pan, Stephen D. Hauschka, J. Patrick Gonzalez, Carl A. Morris, Joel S. Schneider, Dongsheng Duan, Jeffrey S. Chamberlain, Barry J. Byrne, Joe. N. Kornegay
Publikováno v:
Science translational medicine. 15(677)
Duchenne muscular dystrophy (DMD) is a progressive muscle wasting disease caused by the absence of dystrophin, a membrane-stabilizing protein encoded by the DMD gene. Although mouse models of DMD provide insight into the potential of a corrective the
Autor:
Ramarao Vepachedu, Trevor Broadt, Peter Pechan, Brian Bowser, Nora Yang, George Mitra, Asaf Alimardanov, Samir Hussein Shaban, Jenna Burns, Nagarathinam Selvaraj, Chao-Kuei Wang, Nirmala Saptharishi, Diane Golebiowski
Publikováno v:
Hum Gene Ther
Recombinant adeno-associated viral (rAAV) vector-based gene therapy has been adapted for use in more than 100 clinical trials. This is mainly because of its excellent safety profile, ability to target a wide range of tissues, stable transgene express
Autor:
Diane Golebiowski, Marissa Stanvick
Publikováno v:
Cell and Gene Therapy Insights. 8:793-793