Zobrazeno 1 - 10
of 262
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:
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
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:
Rikke Hahn Kofoed, Stefan Heinen, Joseph Silburt, Sonam Dubey, Chinaza Lilian Dibia, Miriam Maes, Elizabeth M. Simpson, Kullervo Hynynen, Isabelle Aubert
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 23, Iss , Pp 390-405 (2021)
Efficient disease-modifying treatments for Alzheimer disease, the most common form of dementia, have yet to be established. Gene therapy has the potential to provide the long-term production of therapeutic in the brain following a single administrati
Externí odkaz:
https://doaj.org/article/2fac79d9eff6473f8934f1dbcffeddbc
Autor:
Snyder, Charles M.
Publikováno v:
New York History, 1950 Apr 01. 31(2), 200-201.
Externí odkaz:
https://www.jstor.org/stable/23149782
Autor:
Seyedeh Zeinab Mirjalili Mohanna, Jack W. Hickmott, Siu Ling Lam, Nina Y. Chiu, Tess C. Lengyell, Beatrice M. Tam, Orson L. Moritz, Elizabeth M. Simpson
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 17, Iss , Pp 478-490 (2020)
Aniridia is a rare eye disorder, which is caused by mutations in the paired box 6 (PAX6) gene and results in vision loss due to the lack of a long-term vision-saving therapy. One potential approach to treating aniridia is targeted CRISPR-based genome
Externí odkaz:
https://doaj.org/article/72def171bf954da9a10a19c51103507a
Autor:
Terri L. Petkau, Benjamin Life, Ge Lu, Jasmine Yang, Oriol Fornes, Wyeth Wasserman, Elizabeth M. Simpson, Blair R. Leavitt
Publikováno v:
Neurobiology of Disease, Vol 153, Iss , Pp 105314- (2021)
The granulin protein (also known as, and hereafter referred to as, progranulin) is a secreted glycoprotein that contributes to overall brain health. Heterozygous loss-of-function mutations in the gene encoding the progranulin protein (Granulin Precur
Externí odkaz:
https://doaj.org/article/a805011b199a45218a7ae5bae9f66a93
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
Publikováno v:
Gene Therapy.
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::ce9fbefafd9c8e29f30e179bb77adc5a
https://doi.org/10.1038/s41434-023-00389-y
https://doi.org/10.1038/s41434-023-00389-y