Zobrazeno 1 - 10
of 184
pro vyhledávání: '"Elizabeth M Simpson"'
Autor:
Alissandra de Moura Gomes, Terri L. Petkau, Andrea J. Korecki, Oriol Fornes, Adriana Galvan, Ge Lu, Austin M. Hill, Siu Ling Lam, Anqi Yao, Rachelle A. Farkas, Wyeth W. Wasserman, Yoland Smith, Elizabeth M. Simpson, Blair R. Leavitt
Publikováno v:
Scientific Reports, Vol 14, Iss 1, Pp 1-19 (2024)
Abstract Compact cell type-specific promoters are important tools for basic and preclinical research and clinical delivery of gene therapy. In this work, we designed novel MiniPromoters to target D1 and D2 type dopaminoceptive medium spiny neurons in
Externí odkaz:
https://doaj.org/article/ccbb413e18624dfaadafe59f90e06ce7
Autor:
Marisa Connell, Helen Chen, Jihong Jiang, Chia-Wei Kuan, Abbas Fotovati, Tony LH Chu, Zhengcheng He, Tess C Lengyell, Huaibiao Li, Torsten Kroll, Amanda M Li, Daniel Goldowitz, Lucien Frappart, Aspasia Ploubidou, Millan S Patel, Linda M Pilarski, Elizabeth M Simpson, Philipp F Lange, Douglas W Allan, Christopher A Maxwell
Publikováno v:
eLife, Vol 6 (2017)
Oriented cell division is one mechanism progenitor cells use during development and to maintain tissue homeostasis. Common to most cell types is the asymmetric establishment and regulation of cortical NuMA-dynein complexes that position the mitotic s
Externí odkaz:
https://doaj.org/article/16ea9b4f47284197a021ef8d519f1ea6
Autor:
Jack W Hickmott, Chih-yu Chen, David J Arenillas, Andrea J Korecki, Siu Ling Lam, Laurie L Molday, Russell J Bonaguro, Michelle Zhou, Alice Y Chou, Anthony Mathelier, Sanford L Boye, William W Hauswirth, Robert S Molday, Wyeth W Wasserman, Elizabeth M Simpson
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 3, Iss C (2016)
Current gene therapies predominantly use small, strong, and readily available ubiquitous promoters. However, as the field matures, the availability of small, cell-specific promoters would be greatly beneficial. Here we design seven small promoters fr
Externí odkaz:
https://doaj.org/article/ec3b5f27a8ba4237bd6536d98ddff289
Autor:
Charles N de Leeuw, Frank M Dyka, Sanford L Boye, Stéphanie Laprise, Michelle Zhou, Alice Y Chou, Lisa Borretta, Simone C McInerny, Kathleen G Banks, Elodie Portales-Casamar, Magdalena I Swanson, Cletus A D'Souza, Shannon E Boye, Steven JM Jones, Robert A Holt, Daniel Goldowitz, William W Hauswirth, Wyeth W Wasserman, Elizabeth M Simpson
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 1, Iss C (2014)
Critical for human gene therapy is the availability of small promoters tools to drive gene expression in a highly specific and reproducible manner. We tackled this challenge by developing human DNA MiniPromoters (MiniPs) using computational biology a
Externí odkaz:
https://doaj.org/article/a4992868fae84ac68f19247787a4fd6f
Autor:
Elisa M. Crombie, Andrea J. Korecki, Karen Cleverley, Bethany A. Adair, Thomas J. Cunningham, Weaverly Colleen Lee, Tess C. Lengyell, Cheryl Maduro, Victor Mo, Liam M. Slade, Ines Zouhair, Elizabeth M. C. Fisher, Elizabeth M. Simpson
Publikováno v:
Disease Models & Mechanisms, Vol 17, Iss 7 (2024)
Externí odkaz:
https://doaj.org/article/9121673d743d4e96a21019e14ea29d93
Publikováno v:
PLoS Genetics, Vol 8, Iss 3, p e1002544 (2012)
An increasing body of literature from genome-wide association studies and human whole-genome sequencing highlights the identification of large numbers of candidate regulatory variants of potential therapeutic interest in numerous diseases. Our relati
Externí odkaz:
https://doaj.org/article/233a496debe64c12aad436a8d36af29b
Autor:
Byron Yu-Lin Kuo, Ying Chen, Slavita Bohacec, Ojvind Johansson, Wyeth W Wasserman, Elizabeth M Simpson
Publikováno v:
PLoS Computational Biology, Vol 2, Iss 4, p e34 (2006)
Serial analysis of gene expression (SAGE) not only is a method for profiling the global expression of genes, but also offers the opportunity for the discovery of novel transcripts. SAGE tags are mapped to known transcripts to determine the gene of or
Externí odkaz:
https://doaj.org/article/1e37d59f178347f38c8e7805011bd15d
Autor:
Bethany A. Adair, Andrea J. Korecki, Diana Djaksigulova, Pamela K. Wagner, Nina Y. Chiu, Siu Ling Lam, Tess C. Lengyell, Blair R. Leavitt, Elizabeth M. Simpson
Publikováno v:
Ophthalmology and Therapy, Vol 12, Iss 4, Pp 2049-2068 (2023)
Abstract Introduction Aniridia is a rare congenital vision-loss disease caused by heterozygous variants in the PAX6 gene. There is no vision-saving therapy, but one exciting approach is to use CRISPR/Cas9 to permanently correct the causal genomic var
Externí odkaz:
https://doaj.org/article/236a9d7004ac47589a17158051a80833
Autor:
Seyedeh Zeinab Mirjalili Mohanna, Diana Djaksigulova, Austin M. Hill, Pamela K. Wagner, Elizabeth M. Simpson, Blair R. Leavitt
Publikováno v:
Journal of Controlled Release. 350:401-413
CRISPR/Cas9-based genome-editing therapies are poised to change the clinical outcome for many diseases with validated therapeutic targets awaiting an appropriate delivery system. Recent advances in lipid nanoparticle (LNP) technology make them an att
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::8df233d41b63df7dad159771327d705d
https://doi.org/10.1016/j.jconrel.2022.08.042
https://doi.org/10.1016/j.jconrel.2022.08.042
Publikováno v:
Gene Therapy.
Recently safety concerns have been raised in connection with high doses of recombinant adeno-associated viruses (rAAV). Therefore, we undertook a series of experiments to test viral capsid (rAAV9 and rAAV-PHP.B), dose, and route of administration (in
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::b010fd5ca6c9dda9a0852f820d94774f
https://doi.org/10.1038/s41434-023-00400-6
https://doi.org/10.1038/s41434-023-00400-6