Zobrazeno 1 - 10
of 117
pro vyhledávání: '"David R. Hampson"'
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 28, Iss , Pp 330-343 (2023)
Impairment of GABAergic inhibitory neuronal function is linked to epilepsy and other neurological and psychiatric disorders. Recombinant adeno-associated virus (rAAV)-based gene therapy targeting GABAergic neurons is a promising treatment for GABA-as
Externí odkaz:
https://doaj.org/article/633f6c85c70a407c989042e89a913a96
Autor:
Hayes Wong, Alexander W.M. Hooper, Hye Ri Kang, Shiron J. Lee, Jiayi Zhao, Chanchal Sadhu, Satinder Rawat, Steven J. Gray, David R. Hampson
Publikováno v:
JCI Insight, Vol 8, Iss 11 (2023)
Fragile X syndrome is a neurodevelopmental disorder caused by the absence of the mRNA-binding protein fragile X messenger ribonucleoprotein (FMRP). Because FMRP is a highly pleiotropic protein controlling the expression of hundreds of genes, viral ve
Externí odkaz:
https://doaj.org/article/7f55ad7e4b43445b8fa490f3c56b476a
Autor:
Alexander W.M. Hooper, Hayes Wong, Yosuke Niibori, Rozita Abdoli, Subha Karumuthil-Melethil, Chunping Qiao, Olivier Danos, Joseph T. Bruder, David R. Hampson
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 22, Iss , Pp 196-209 (2021)
Fragile X syndrome (FXS), a neurodevelopmental disorder with no known cure, is caused by a lack of expression of the fragile X mental retardation protein (FMRP). As a single-gene disorder, FXS is an excellent candidate for viral-vector-based gene the
Externí odkaz:
https://doaj.org/article/39f2762e4bc2409e975a474aed179042
Publikováno v:
Frontiers in Neurology, Vol 12 (2021)
Several neurological and psychiatric disorders have been associated with impairments in GABAergic inhibitory neurons in the brain. Thus, in the current era of accelerated development of molecular medicine and biologically-based drugs, there is a need
Externí odkaz:
https://doaj.org/article/6efa64b71cd14295b4e1a8a6bf4a6dca
Autor:
Hayes Wong, Alexander W.M. Hooper, Yosuke Niibori, Shiron J. Lee, Luca A. Hategan, Liang Zhang, Subha Karumuthil-Melethil, Sally M. Till, Peter C. Kind, Olivier Danos, Joseph T. Bruder, David R. Hampson
Publikováno v:
Neurobiology of Disease, Vol 146, Iss , Pp 105118- (2020)
Fragile X syndrome (FXS), a neurodevelopmental disorder with autistic features, is caused by the loss of the fragile X mental retardation protein. Sex-specific differences in the clinical profile have been observed in FXS patients, but few studies ha
Externí odkaz:
https://doaj.org/article/408689585b4c4ab0b7a92f55b5b78196
The Application of Adeno-Associated Viral Vector Gene Therapy to the Treatment of Fragile X Syndrome
Publikováno v:
Brain Sciences, Vol 9, Iss 2, p 32 (2019)
Viral vector-mediated gene therapy has grown by leaps and bounds over the past several years. Although the reasons for this progress are varied, a deeper understanding of the basic biology of the viruses, the identification of new and improved versio
Externí odkaz:
https://doaj.org/article/cd8bf902289f484994e02e596bb93686
Autor:
Roman Pekhletski, David R. Hampson
Publikováno v:
BioTechniques, Vol 20, Iss 6, Pp 956-960 (1996)
Externí odkaz:
https://doaj.org/article/07e0dd3b65f9450f9f5787792aafb21c
Autor:
Rozita Abdoli, Subha Karumuthil-Melethil, Yosuke Niibori, Alexander W.M. Hooper, David R. Hampson, Olivier Danos, Hayes Wong, Chunping Qiao, Joseph T. Bruder
Publikováno v:
Molecular Therapy. Methods & Clinical Development
Molecular Therapy: Methods & Clinical Development, Vol 22, Iss, Pp 196-209 (2021)
Molecular Therapy: Methods & Clinical Development, Vol 22, Iss, Pp 196-209 (2021)
Fragile X syndrome (FXS), a neurodevelopmental disorder with no known cure, is caused by a lack of expression of the fragile X mental retardation protein (FMRP). As a single-gene disorder, FXS is an excellent candidate for viral-vector-based gene the
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::57bc7c918cc34d19f59dd4201f282537
https://doi.org/10.1016/j.omtm.2021.06.013
https://doi.org/10.1016/j.omtm.2021.06.013
Autor:
Lu-Yang Wang, Tian Kong, David R. Hampson, Enea Koxhioni, Yosuke Niibori, James H. Eubanks, Alexander W.M. Hooper, Shervin Gholizadeh, Jason Arsenault, Laura K. K. Pacey
Publikováno v:
Human Molecular Genetics
Several X-linked neurodevelopmental disorders including Rett syndrome, induced by mutations in the MECP2 gene, and fragile X syndrome (FXS), caused by mutations in the FMR1 gene, share autism-related features. The mRNA coding for methyl CpG binding p
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::c5597bf9877fb975144e909fff1b7f96
https://doi.org/10.1093/hmg/ddaa226
https://doi.org/10.1093/hmg/ddaa226
Publikováno v:
Human Gene Therapy
Dravet syndrome (DS) is a neurodevelopmental genetic disorder caused by mutations in the SCN1A gene encoding the α subunit of the NaV1.1 voltage-gated sodium channel that controls neuronal action potential firing. The high density of this mutated ch
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::7b91f470b88d98a872759bf2b8c223d1
http://europepmc.org/articles/PMC7087406
http://europepmc.org/articles/PMC7087406