Zobrazeno 1 - 10
of 19
pro vyhledávání: '"Megan S. Keiser"'
Autor:
Ellie M. Carrell, Yong Hong Chen, Paul T. Ranum, Stephanie L. Coffin, Larry N. Singh, Luis Tecedor, Megan S. Keiser, Eloise Hudry, Bradley T. Hyman, Beverly L. Davidson
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 33, Iss , Pp 296-304 (2023)
Recombinant adeno-associated viral vectors (rAAVs) are a promising strategy to treat neurodegenerative diseases because of their ability to infect non-dividing cells and confer long-term transgene expression. Despite an ever-growing library of capsid
Externí odkaz:
https://doaj.org/article/adb40a4cec5b4a3d9ab912e739f69a5c
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 25, Iss , Pp 333-343 (2022)
Spinocerebellar ataxia type 1 (SCA1) is an autosomal dominant neurodegenerative disease caused by a (CAG) repeat expansion in the coding sequence of ATXN1. The primary mechanism of disease in SCA1 is toxic gain of function by polyglutamine-expanded m
Externí odkaz:
https://doaj.org/article/63fbde220c9941b89c060e5e66a8214a
Publikováno v:
Neurobiology of Disease, Vol 56, Iss , Pp 6-13 (2013)
Spinocerebellar Ataxia Type 1 (SCA1) is an autosomal dominant late onset neurodegenerative disease caused by an expanded polyglutamine tract in ataxin-1. Here, we compared the protective effects of overexpressing ataxin-1-like using recombinant AAVs,
Externí odkaz:
https://doaj.org/article/b4657bcb5aa94fbd867ab152a83c24e3
Autor:
Paul T. Ranum, Luis Tecedor, Megan S. Keiser, Yong Hong Chen, David E. Leib, Xueyuan Liu, Beverly L. Davidson
Publikováno v:
Molecular therapy : the journal of the American Society of Gene Therapy.
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::8cb29cedc4333ee9797d74667b78be11
https://pubmed.ncbi.nlm.nih.gov/36610400
https://pubmed.ncbi.nlm.nih.gov/36610400
Autor:
Pedro Gonzalez-Alegre, Yong Hong Chen, Geary R. Smith, Enrico Radaelli, Joel M. Stein, Timothy J. Lucas, Megan S. Keiser, Beverly L. Davidson, Ellie M. Carrell, Ronald L. Wolf, Amy Muehlmatt, Carolyn M. Yrigollen, Paul T. Ranum
Publikováno v:
Nat Med
RNA interference (RNAi) for spinocerebellar ataxia type 1 can prevent and reverse behavioral deficits and neuropathological readouts in mouse models, with safety and benefit lasting over many months. The RNAi trigger, expressed from adeno-associated
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::a98a56cafb5ddb46197fe5fbde1961fc
https://europepmc.org/articles/PMC8605996/
https://europepmc.org/articles/PMC8605996/
Publikováno v:
Molecular Therapy
Huntington disease (HD) is a fatal dominantly inherited neurodegenerative disorder caused by CAG repeat expansion (>36 repeats) within the first exon of the huntingtin gene. Although mutant huntingtin (mHTT) is ubiquitously expressed, the brain shows
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::35bd26e10699b5f267ead7cffcb60c91
http://europepmc.org/articles/PMC5363210
http://europepmc.org/articles/PMC5363210
Publikováno v:
Current protocols in mouse biology. 8(4)
Adeno-associated virus (AAV) vectors are exemplary tools for studying gene function in vivo and are particularly favorable for transferring genes of interest into brain tissues. They have shown great promise as a gene therapy vector for preclinical a
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::ad7866fd6dd08ddec306e2255e14f3f3
https://pubmed.ncbi.nlm.nih.gov/30489697
https://pubmed.ncbi.nlm.nih.gov/30489697
Publikováno v:
Current protocols in mouse biology. 8(4)
Stereotaxic intracranial injection of viral vectors is a valuable technique to directly deliver genetic material to a specific population of cells in the central nervous system of a mouse model. This enables scientists to test candidate gene therapie
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::ff933a797d24abd1b549b9cc93207e9d
https://pubmed.ncbi.nlm.nih.gov/30395418
https://pubmed.ncbi.nlm.nih.gov/30395418
Autor:
Congsheng Cheng, Alex Mas Monteys, Megan S. Keiser, Ryan M. Spengler, Beverly L. Davidson, Julianne M. Rieders, Shyam Ramachandran
Publikováno v:
Human Molecular Genetics
Polyglutamine (polyQ) repeat diseases are a class of neurodegenerative disorders caused by CAG-repeat expansion. There are diverse cellular mechanisms behind the pathogenesis of polyQ disorders, including transcriptional dysregulation. Interestingly,
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::c2be00c739339d2b0e4c0ce4b27db53c
https://doi.org/10.1093/hmg/ddy331
https://doi.org/10.1093/hmg/ddy331
Publikováno v:
Human Molecular Genetics. 25:R53-R64
RNA-targeting approaches are emerging as viable therapeutics that offer an alternative method to modulate traditionally ‘undrugable’ targets. In the case of dominantly inherited neurodegenerative diseases, gene suppression strategies can target t
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::683489c809beac64391816f1cc449431
https://doi.org/10.1093/hmg/ddv442
https://doi.org/10.1093/hmg/ddv442