Zobrazeno 1 - 10
of 52
pro vyhledávání: '"Kelly M Podetz-Pedersen"'
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 5, Iss C (2016)
The Sleeping Beauty (SB) transposon system has been shown to enable long-term gene expression by integrating new sequences into host cell chromosomes. We found that the recently reported SB100x hyperactive transposase conferred a surprisingly high le
Externí odkaz:
https://doaj.org/article/98ab76a7af2242b09d9c7c6e8115eb9e
Autor:
Jesse D Riordan, Vincent W Keng, Barbara R Tschida, Todd E Scheetz, Jason B Bell, Kelly M Podetz-Pedersen, Catherine D Moser, Neal G Copeland, Nancy A Jenkins, Lewis R Roberts, David A Largaespada, Adam J Dupuy
Publikováno v:
PLoS Genetics, Vol 9, Iss 4, p e1003441 (2013)
We previously utilized a Sleeping Beauty (SB) transposon mutagenesis screen to discover novel drivers of HCC. This approach identified recurrent mutations within the Dlk1-Dio3 imprinted domain, indicating that alteration of one or more elements withi
Externí odkaz:
https://doaj.org/article/74ed0ae8a4344f6fbb931f1bfaf4706b
Autor:
Kanut Laoharawee, Kelly M. Podetz-Pedersen, Tam T. Nguyen, Sajya M. Singh, Miles C. Smith, Lalitha R. Belur, Walter C. Low, Karen F. Kozarsky, R. Scott McIvor
Publikováno v:
Molecular Genetics and Metabolism Reports, Vol 34, Iss , Pp 100956- (2023)
Hunter syndrome is a rare x-linked recessive genetic disorder that affects lysosomal metabolism due to deficiency of iduronate-2-sulfatase (IDS), with subsequent accumulation of glycosaminoglycans heparan and dermatan sulfates (GAG). Enzyme replaceme
Externí odkaz:
https://doaj.org/article/844641009ad346d0b12f5b18be323ac8
Autor:
Lalitha R. Belur, Megan Romero, Junggu Lee, Kelly M. Podetz-Pedersen, Zhenhong Nan, Maureen S. Riedl, Lucy Vulchanova, Kelley F. Kitto, Carolyn A. Fairbanks, Karen F. Kozarsky, Paul J. Orchard, William H. Frey, Walter C. Low, R. Scott McIvor
Publikováno v:
Frontiers in Molecular Neuroscience, Vol 14 (2021)
Mucopolysaccharidosis type I (MPS I) is an inherited metabolic disorder caused by deficiency of the lysosomal enzyme alpha-L-iduronidase (IDUA). The two current treatments [hematopoietic stem cell transplantation (HSCT) and enzyme replacement therapy
Externí odkaz:
https://doaj.org/article/612b20e3a1914626b04655208aae2b85
Autor:
Kelly M. Podetz-Pedersen, Kanut Laoharawee, Sajya Singh, Tam T. Nguyen, Miles C. Smith, Alexa Temme, Karen Kozarsky, R. Scott McIvor, Lalitha R. Belur
Publikováno v:
Human Gene Therapy. 34:8-18
The mucopolysaccharidoses (MPS) are a group of recessively inherited conditions caused by deficiency of lysosomal enzymes essential to the catabolism of glycosaminoglycans. MPS I is caused by deficiency of the lysosomal enzyme alpha-L-iduronidase (ID
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::d79d1578844c99aeb3d28ce7862ff36c
https://doi.org/10.1089/hum.2022.162
https://doi.org/10.1089/hum.2022.162
Autor:
Miles C. Smith, Lalitha R. Belur, Andrea D. Karlen, Olivia Erlanson, Kelly M. Podetz-Pedersen, Jessica McKenzie, Jenn Detellis, Khatuna Gagnidze, Geoffrey Parsons, Nicholas Robinson, Shelby Labarre, Saumil Shah, Justin Furcich, Troy C. Lund, Hsing-Chen Tsai, R. Scott McIvor, Melissa Bonner
Publikováno v:
Human Gene Therapy. 33:1279-1292
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::cdfc4a893a7f592726a497c6e2ab15bd
https://doi.org/10.1089/hum.2022.141
https://doi.org/10.1089/hum.2022.141
Autor:
Paul J. Orchard, Zhenhong Nan, Karen Kozarsky, Kelley F. Kitto, Kelly M. Podetz-Pedersen, Lalitha R. Belur, Megan Romero, William H. Frey, Lucy Vulchanova, Carolyn A. Fairbanks, Junggu Lee, Maureen S. Riedl, Walter C. Low, R. Scott McIvor
Publikováno v:
Frontiers in Molecular Neuroscience
Frontiers in Molecular Neuroscience, Vol 14 (2021)
Frontiers in Molecular Neuroscience, Vol 14 (2021)
Mucopolysaccharidosis type I (MPS I) is an inherited metabolic disorder caused by deficiency of the lysosomal enzyme alpha-L-iduronidase (IDUA). The two current treatments [hematopoietic stem cell transplantation (HSCT) and enzyme replacement therapy
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::f3ddf81503b84f42e3edf829e5e43af1
https://doi.org/10.3389/fnmol.2021.618360
https://doi.org/10.3389/fnmol.2021.618360
Autor:
Lucy Vulchanova, Thuy An Tran, Lalitha R. Belur, Nathaniel M. Singh, Joshua A. Mesick, Karen Kozarsky, R. Scott McIvor, Kelly M. Podetz-Pedersen, Maureen S. Riedl
Publikováno v:
Molecular Genetics and Metabolism Reports, Vol 24, Iss, Pp 100604-(2020)
Molecular Genetics and Metabolism Reports
Molecular Genetics and Metabolism Reports
Mucopolysaccharidosis type I (MPS I) is an inherited metabolic disorder caused by deficiency of alpha-L-iduronidase (IDUA), resulting in accumulation of heparan and dermatan sulfate glycosaminoglycans (GAGs). Individuals with the most severe form of
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::0108e519a64e955fae50a3bcbae768cb
https://pubmed.ncbi.nlm.nih.gov/32461912
https://pubmed.ncbi.nlm.nih.gov/32461912
Autor:
Susan Tom, Kathleen Meyer, Kelly M. Podetz-Pedersen, Renee Cooksley, Michael C. Holmes, R. Scott McIvor, Russell Dekelver, Brenda Koniar, Michelle Rohde, Kanut Laoharawee, Scott Sproul, Chester B. Whitley, Susan St Martin, Robert Radeke, Li Ou, Yolanda Santiago, Thomas Wechsler, Michelle J. Przybilla
Publikováno v:
Molecular Therapy
Mucopolysaccharidosis type I (MPS I) is a severe disease due to deficiency of the lysosomal hydrolase α-L-iduronidase (IDUA) and the subsequent accumulation of the glycosaminoglycans (GAG), leading to progressive, systemic disease and a shortened li
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::056906ea953e0f6ee7a887c3a7320cdb
http://europepmc.org/articles/PMC6319315
http://europepmc.org/articles/PMC6319315
Autor:
Kanut Laoharawee, R. Scott McIvor, Hoang Oanh Nguyen, Michelle Rohde, Robert Radeke, Tam T Nguyen, Michael C. Holmes, Li Ou, Kelly M. Podetz-Pedersen, Scott Sproul, Thomas Wechsler, Russell Dekelver, Chester B. Whitley, Susan St Martin, Susan Tom, Kathleen Meyer
Publikováno v:
Molecular Therapy
Mucopolysaccharidosis type II (MPS II) is an X-linked recessive lysosomal disorder caused by deficiency of iduronate 2-sulfatase (IDS), leading to accumulation of glycosaminoglycans (GAGs) in tissues of affected individuals, progressive disease, and
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::908d268feb85f0ef2ec1b779befa440d
https://doi.org/10.1016/j.ymthe.2018.03.002
https://doi.org/10.1016/j.ymthe.2018.03.002