Zobrazeno 1 - 10
of 376
pro vyhledávání: '"Beverly L. Davidson"'
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 35, Iss 4, Pp 102317- (2024)
Spinocerebellar ataxia type 1 (SCA1) is an adult-onset neurodegenerative disease caused by an expansion of the CAG repeat region of the ATXN1 gene. Currently there are no disease-modifying treatments; however, previous work has shown the potential of
Externí odkaz:
https://doaj.org/article/29e2bfbcf05e4ad1ba3e0ed38d7d2a4d
Autor:
Xueyuan Liu, Riffard Jean-Gilles, Julia Baginski, Christina Cai, Ruilan Yan, Lili Zhang, Kevin Lance, Johannes C.M. van der Loo, Beverly L. Davidson
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 32, Iss 3, Pp 101298- (2024)
Recombinant adeno-associated virus (rAAV)-based gene therapies are expanding in their application. Despite progress in manufacturing, current analytical methods for product quantification and characterization remain largely unchanged. Although critic
Externí odkaz:
https://doaj.org/article/2f77cd4b3e5c4b8fb79a86dae4ba5380
Autor:
Jesse A. Weber, Jonathan F. Lang, Ellie M. Carrell, Mohamad-Gabriel Alameh, Beverly L. Davidson
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 35, Iss 2, Pp 102172- (2024)
Clinical application of CRISPR-Cas9 technology for large deletions of somatic mutations is inefficient, and methods to improve utility suffer from our inability to rapidly assess mono- vs. biallelic deletions. Here we establish a model system for inv
Externí odkaz:
https://doaj.org/article/037b3f7b70b14bb5bc5a44156a339446
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
Mapping PTBP2 binding in human brain identifies SYNGAP1 as a target for therapeutic splice switching
Autor:
Jennine M. Dawicki-McKenna, Alex J. Felix, Elisa A. Waxman, Congsheng Cheng, Defne A. Amado, Paul T. Ranum, Alexey Bogush, Lea V. Dungan, Jean Ann Maguire, Alyssa L. Gagne, Elizabeth A. Heller, Deborah L. French, Beverly L. Davidson, Benjamin L. Prosser
Publikováno v:
Nature Communications, Vol 14, Iss 1, Pp 1-20 (2023)
Abstract Alternative splicing of neuronal genes is controlled partly by the coordinated action of polypyrimidine tract binding proteins (PTBPs). While PTBP1 is ubiquitously expressed, PTBP2 is predominantly neuronal. Here, we define the PTBP2 footpri
Externí odkaz:
https://doaj.org/article/8fbfb46edfea4a479ef94d2853340d5a
Autor:
Li Fang, Alex Mas Monteys, Alexandra Dürr, Megan Keiser, Congsheng Cheng, Akhil Harapanahalli, Pedro Gonzalez-Alegre, Beverly L. Davidson, Kai Wang
Publikováno v:
HGG Advances, Vol 4, Iss 3, Pp 100212- (2023)
Externí odkaz:
https://doaj.org/article/5b6636653ade417b971e1b36bcf3f0fe
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:
Genome Biology, Vol 23, Iss 1, Pp 1-27 (2022)
Abstract Despite recent improvements in basecalling accuracy, nanopore sequencing still has higher error rates on short-tandem repeats (STRs). Instead of using basecalled reads, we developed DeepRepeat which converts ionic current signals into red-gr
Externí odkaz:
https://doaj.org/article/cd5ecec98d6b44debd6abf2177f51d0a
Autor:
Caroline Gubser Keller, Youngah Shin, Alex Mas Monteys, Nicole Renaud, Martin Beibel, Natalia Teider, Thomas Peters, Thomas Faller, Sophie St-Cyr, Judith Knehr, Guglielmo Roma, Alejandro Reyes, Marc Hild, Dmitriy Lukashev, Diethilde Theil, Natalie Dales, Jang-Ho Cha, Beth Borowsky, Ricardo Dolmetsch, Beverly L. Davidson, Rajeev Sivasankaran
Publikováno v:
Nature Communications, Vol 13, Iss 1, Pp 1-11 (2022)
Huntington’s disease (HD) results from the abnormal expansion of CAG repeats in exon 1 of the HTT gene. Here, the authors show that orally available, brain penetrant molecule branaplam lowers HTT transcript by promoting inclusion of a poison exon o
Externí odkaz:
https://doaj.org/article/917384fbd2e34e4887d645320e87dd19
Autor:
Sophie St-Cyr, Daniel D Child, Emilie Giaime, Alicia R Smith, Christine J Pascua, Seung Hahm, Eddine Saiah, Beverly L Davidson
Publikováno v:
PLoS ONE, Vol 17, Iss 8, p e0273710 (2022)
Huntington's Disease (HD) is a dominantly inherited neurodegenerative disease for which the major causes of mortality are neurodegeneration-associated aspiration pneumonia followed by cardiac failure. mTORC1 pathway perturbations are present in HD mo
Externí odkaz:
https://doaj.org/article/5daa31f88c0a405a978f93c37b33aa4c