Zobrazeno 1 - 10
of 57
pro vyhledávání: '"Marco A. Passini"'
Autor:
Li Gan, Leslie C.L. Wu, Jenna A. Wood, Monica Yao, Chris M. Treleaven, Nelsa L. Estrella, Bruce M. Wentworth, Gunnar J. Hanson, Marco A. Passini
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 30, Iss , Pp 17-27 (2022)
Antisense RNA technology is a strategy for the treatment of Duchenne muscular dystrophy (DMD), a progressive and universally fatal X-linked neuromuscular disease caused by frameshift mutations in the gene encoding dystrophin. Phosphorodiamidate morph
Externí odkaz:
https://doaj.org/article/bddb1e4b922e426fa81d57fe314dc534
Autor:
Li-Kai Tsai, Yi-Chun Chen, Wei-Cheng Cheng, Chen-Hung Ting, James C. Dodge, Wuh-Liang Hwu, Seng H. Cheng, Marco A. Passini
Publikováno v:
Neurobiology of Disease, Vol 45, Iss 1, Pp 272-279 (2012)
The efficacy of administering a recombinant adeno-associated virus (AAV) vector encoding human IGF-1 (AAV2/1-hIGF-1) into the deep cerebellar nucleus (DCN) of a type III SMA mouse model was evaluated. High levels of IGF-1 transcripts and protein were
Externí odkaz:
https://doaj.org/article/6e84d4b8c4404d04ba18b970861eb675
Publikováno v:
PLoS ONE, Vol 3, Iss 10, p e3468 (2008)
RNA modalities are developing as a powerful means to re-direct pathogenic pre-mRNA splicing events. Improving the efficiency of these molecules in vivo is critical as they move towards clinical applications. Spinal muscular atrophy (SMA) is caused by
Externí odkaz:
https://doaj.org/article/5226e55379bc40b0a1f1020f54a50ae2
Autor:
Chen-Hung Ting, Chien-Lin Chen, Wuh-Liang Hwu, Seng H. Cheng, Sue Lin-Chao, Marco A. Passini, James Dodge, Li-Kai Tsai
Publikováno v:
Molecular Therapy. 22(8):1450-1459
Spinal muscular atrophy is a progressive motor neuron disease caused by a deficiency of survival motor neuron. In this study, we evaluated the efficacy of intravenous administration of a recombinant adeno-associated virus (AAV1) vector encoding human
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::bf59704a6c97c7a95b5d91713bff6538
Autor:
Karen M. Ashe, Jie Bu, Marco A. Passini, Edward H. Schuchman, John Bringas, John Marshall, John Forsayeth, James Dodge, Seng H. Cheng, Lamya S. Shihabuddin, Krystof S. Bankiewicz, Mario A. Cabrera-Salazar
Publikováno v:
Molecular therapy : the journal of the American Society of Gene Therapy, vol 20, iss 10
Niemann-Pick disease Type A (NPA) is a neuronopathic lysosomal storage disease (LSD) caused by the loss of acid sphingomyelinase (ASM). The goals of the current study are to ascertain the levels of human ASM that are efficacious in ASM knockout (ASMK
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::5fbc94f7174d6e5e0cc2fccf581f584f
https://doi.org/10.1038/mt.2012.118
https://doi.org/10.1038/mt.2012.118
Autor:
S. Pablo Sardi, Marco A. Passini, Jie Bu, Lisa Woodworth, Gregory D Hurlbut, James Dodge, Lamya S. Shihabuddin, Christopher M. Treleaven, Thomas J. Tamsett, Jonathan A. Fidler, Seng H. Cheng
Publikováno v:
Molecular Therapy. 20(9):1713-1723
Central nervous system (CNS)-directed gene therapy with recombinant adeno-associated virus (AAV) vectors has been used effectively to slow disease course in mouse models of several neurodegenerative diseases. However, these vectors were typically tes
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::49ee5a62c935de9e9063c0e85666c9ef
Autor:
Steven J. Gray, Richard Jude Samulski, Marco A. Passini, K A Matthews, Chalonda R. Handy, Thais Federici, Jason Taub, Joshua C Grieger, Griffin R. Baum, Nicholas M. Boulis
Publikováno v:
Gene Therapy. 19:852-859
Adeno-associated viral vector 9 (AAV9) has recently been shown to penetrate the blood–brain barrier via intravascular administration, making it a good candidate for diffuse gene delivery. However, the potential side effects of systemic delivery are
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::b7cef131364211af38b1fa3d8dcf78c1
https://doi.org/10.1038/gt.2011.130
https://doi.org/10.1038/gt.2011.130
Autor:
Lamya S. Shihabuddin, S.P. Sardi, Piotr Hadaczek, R.M. Richardson, Massimo S. Fiandaca, E. Aguilar Salegio, Marco A. Passini, John Forsayeth, Krystof S. Bankiewicz, Adrian P. Kells, John Bringas, S H Cheng
Publikováno v:
Human Gene Therapy. 21:1093-1103
Gene replacement therapy for the neurological deficits caused by lysosomal storage disorders, such as in Niemann-Pick disease type A, will require widespread expression of efficacious levels of acid sphingomyelinase (ASM) in the infant human brain. A
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::505811373ff6ec5a279de7ef417f6ed6
https://doi.org/10.1089/hum.2010.040
https://doi.org/10.1089/hum.2010.040
Autor:
Yimin Hua, Kentaro Sahashi, Marco A. Passini, Adrian R. Krainer, Gene Hung, C. Frank Bennett, Frank Rigo
Publikováno v:
Genes & Development. 24:1634-1644
Increasing survival of motor neuron 2, centromeric (SMN2) exon 7 inclusion to express more full-length SMN protein in motor neurons is a promising approach to treat spinal muscular atrophy (SMA), a genetic neurodegenerative disease. Previously, we id
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::d0bc8d7cfcaa3bc9ce65edbe00ebffcb
https://doi.org/10.1101/gad.1941310
https://doi.org/10.1101/gad.1941310
Autor:
Seng H. Cheng, Edward H. Schuchman, Jonathan A. Fidler, Christopher M. Treleaven, Marco A. Passini, Kenneth P. Karey, Denise Griffiths, Wendy Yang, Tatyana V. Taksir, Lamya S. Shihabuddin, James Dodge, Jennifer Clarke
Publikováno v:
Experimental Neurology. 215:349-357
Niemann-Pick A (NPA) disease is a lysosomal storage disorder (LSD) caused by a deficiency in acid sphingomyelinase (ASM) activity. Previously, we showed that the storage pathology in the ASM knockout (ASMKO) mouse brain could be corrected by intracer
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::90be1695519f0d90b468493266c95301
https://doi.org/10.1016/j.expneurol.2008.10.021
https://doi.org/10.1016/j.expneurol.2008.10.021