Zobrazeno 1 - 10
of 41
pro vyhledávání: '"Elena L, Aronovich"'
Publikováno v:
PLoS ONE, Vol 8, Iss 10, p e78161 (2013)
The Sleeping Beauty transposon system, a non-viral, integrating vector that can deliver the alpha-L-iduronidase-encoding gene, is efficient in correcting mucopolysaccharidosis type I in NOD/SCID mice. However, in previous studies we failed to attain
Externí odkaz:
https://doaj.org/article/74230a94deee433a9d3f7d99c71736f8
Autor:
Kendra A. Hyland, R. Scott McIvor, Jason B. Bell, David W. Hunter, Erik R. Olson, Roland Gunther, Perry B. Hackett, Elena L. Aronovich, Myra Rusten
Publikováno v:
Human Gene Therapy. 28:541-550
The Sleeping Beauty transposon system has been extensively tested for integration of reporter and therapeutic genes in vitro and in vivo in mice. Dogs were used as a large animal model for human therapy and minimally invasive infusion of DNA solution
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::b17f61ca01269aded0fc54f5a4897d65
https://doi.org/10.1089/hum.2017.003
https://doi.org/10.1089/hum.2017.003
Ruxolitinib Reduces Endothelial Pro-Adhesive Interactions: Implications for JAK2V617+ MPN Thrombosis
Autor:
David E Reynolds, Angelica DaSilva, Aithanh Nguyen, Julia Nguyen, Joan D. Beckman, Gregory M. Vercellotti, Geneva D. Doak, Elena L. Aronovich, David K. Wood
Publikováno v:
Blood. 136:1-1
Introduction The JAK2V617F+ mutation occurs in up to 95% of patients with polycythemia vera (PV) and increases the risk of thrombosis 6-fold. Recent studies demonstrate that JAK2V617F+ endothelial cells express pro-adhesive proteins, suggesting that
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::aa9960658c61d494888d4a41a2a1ccb7
https://doi.org/10.1182/blood-2020-140116
https://doi.org/10.1182/blood-2020-140116
Autor:
Perry B. Hackett, Elena L. Aronovich
Publikováno v:
Molecular Genetics and Metabolism. 114(2):83-93
Most lysosomal storage disorders affect the nervous system as well as other tissues and organs of the body. Previously, the complexities of these diseases, particularly in treating neurologic abnormalities, were too great to surmount. However, based
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::1937e15ad9b5648a84919325d90cd893
Autor:
Elena L. Aronovich, Myra Rusten, Jason B. Bell, Bryan C. Hall, David W. Hunter, N. Matthew Ellinwood, R. Scott McIvor, Erik R. Olson, Perry B. Hackett, Kendra A. Hyland
Publikováno v:
Human gene therapy. 28(7)
The non-viral, integrating Sleeping Beauty (SB) transposon system is efficient in treating systemic monogenic disease in mice, including hemophilia A and B caused by deficiency of blood clotting factors and mucopolysaccharidosis types I and VII cause
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::75f921ce5b196844fd855506cec4e681
https://pubmed.ncbi.nlm.nih.gov/28719250
https://pubmed.ncbi.nlm.nih.gov/28719250
Autor:
Jeffrey M. Schreifels, Irina V. Nesmelova, Claire E. Carpentier, Elena L. Aronovich, Daniel F. Carlson, Perry B. Hackett
Publikováno v:
Protein Science. 23:23-33
The Sleeping Beauty (SB) transposon is the most widely used DNA transposon in genetic applications and is the only DNA transposon thus far in clinical trials for human gene therapy. In the absence of atomic level structural information, the developme
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::73536a9a8fa5a36994e6df9b351d00cc
https://doi.org/10.1002/pro.2386
https://doi.org/10.1002/pro.2386
Autor:
Leah R. Hanson, R. Scott McIvor, Elena L. Aronovich, Daniel A. Wolf, Zhenhong Nan, Walter C. Low, William H. Frey
Publikováno v:
Molecular Genetics and Metabolism. 106:131-134
Here we provide the first evidence that therapeutic levels of a lysosomal enzyme can bypass the blood-brain barrier following intranasal administration. α-L-iduronidase (IDUA) activity was detected throughout the brains of IDUA-deficient mice follow
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::023a09b2310583a58029cfea72dc31b5
https://doi.org/10.1016/j.ymgme.2012.02.006
https://doi.org/10.1016/j.ymgme.2012.02.006
Autor:
Jason B. Bell, R. Scott McIvor, Perry B. Hackett, Laurence J.N. Cooper, Steven J. Kass, Elena L. Aronovich, David J. Hunter, Myra Urness
Publikováno v:
Current Gene Therapy. 11:341-349
Sleeping Beauty (SB) transposons have been effective in delivering therapeutic genes to treat certain diseases in mice. Hydrodynamic gene delivery of integrating transposons to 5–20% of the hepatocytes in a mouse results in persistent elevated expr
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::12414841fdc7a367de6aef23b5858169
https://doi.org/10.2174/156652311797415827
https://doi.org/10.2174/156652311797415827
Autor:
Erik R. Olson, Perry B. Hackett, Bruce R. Blazar, R. Scott McIvor, Kendra A. Hyland, Karl J. Clark, Jakub Tolar, Elena L. Aronovich
Publikováno v:
The Journal of Gene Medicine. 13:462-469
Background The Sleeping Beauty (SB) transposon system can insert defined sequences into chromosomes to direct the extended expression of therapeutic genes. Our goal is to develop the SB system for nonviral complementation of Fanconi anemia (FA), a ra
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::6133d6fc42a7d56f11d4c8c829bcf32f
https://doi.org/10.1002/jgm.1589
https://doi.org/10.1002/jgm.1589
Publikováno v:
Human Molecular Genetics. 20:R14-R20
Over the past decade, the Sleeping Beauty (SB) transposon system has been developed as the leading non-viral vector for gene therapy. This vector combines the advantages of viruses and naked DNA. Here we review progress over the last 2 years in vecto
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::1a635fe9ea67dacc8aa0463ab618f032
https://doi.org/10.1093/hmg/ddr140
https://doi.org/10.1093/hmg/ddr140