Zobrazeno 1 - 10
of 36
pro vyhledávání: '"Jenny A. Greig"'
Autor:
Jenny A. Greig, Joanna K. Chorazeczewski, Vivek Chowdhary, Melanie K. Smith, Matthew Jennis, James C. Tarrant, Elizabeth L. Buza, Kimberly Coughlan, Paolo G.V. Martini, James M. Wilson
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 29, Iss , Pp 32-39 (2023)
Crigler-Najjar syndrome is a rare disorder of bilirubin metabolism caused by uridine diphosphate glucuronosyl transferase 1A1 (UGT1A1) mutations characterized by hyperbilirubinemia and jaundice. No cure currently exists; treatment options are limited
Externí odkaz:
https://doaj.org/article/4c4c351c175a4e80928fe2e172b5b2ec
Autor:
Makoto Horiuchi, Christian J. Hinderer, Jenny A. Greig, Cecilia Dyer, Elizabeth L. Buza, Peter Bell, Jessica A. Chichester, Peter M. Hayashi, Hanying Yan, Tamara Goode, James M. Wilson
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 27, Iss , Pp 272-280 (2022)
Gene therapy using neurotropic adeno-associated virus vectors represents an emerging solution for genetic disorders affecting the central nervous system. The first approved central nervous system-targeting adeno-associated virus gene therapy, Zolgens
Externí odkaz:
https://doaj.org/article/47d10615a3624fa0872c118998bf302a
Publikováno v:
BMC Genomics, Vol 21, Iss 1, Pp 1-12 (2020)
Abstract Background Identifying nuclease-induced double-stranded breaks in DNA on a genome-wide scale is critical for assessing the safety and efficacy of genome editing therapies. We previously demonstrated that after administering adeno-associated
Externí odkaz:
https://doaj.org/article/84dd2ff60759418d8a28d6e3f281d5ef
Publikováno v:
BMC Genomics, Vol 21, Iss 1, Pp 1-3 (2020)
An amendment to this paper has been published and can be accessed via the original article.
Externí odkaz:
https://doaj.org/article/71d102379f5c4af1a400acaff58b14b9
Autor:
Jenny A. Greig, Roberto Calcedo, Leticia Kuri-Cervantes, Jayme M.L. Nordin, Jessica Albrecht, Erin Bote, Tamara Goode, Edward A. Chroscinski, Peter Bell, Laura K. Richman, Michael R. Betts, James M. Wilson
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 11, Iss , Pp 191-201 (2018)
Systemic delivery of adeno-associated viral (AAV) vectors has been evaluated for the treatment of several liver diseases, including homozygous familial hypercholesterolemia, ornithine transcarbamylase deficiency, and hemophilia. Here, we evaluated th
Externí odkaz:
https://doaj.org/article/01118e8f567e49fb8ab1d3079ca6e907
Autor:
Jenny A. Greig, Jayme M.L. Nordin, Christine Draper, Deirdre McMenamin, Edward A. Chroscinski, Peter Bell, John T. Gray, Laura K. Richman, James M. Wilson
Publikováno v:
Molecular Therapy: Methods & Clinical Development, Vol 10, Iss , Pp 237-244 (2018)
Liver metabolism disorders are attractive targets for gene therapy, because low vector doses can reverse the buildup of toxic metabolites in the blood. Crigler-Najjar syndrome is an inherited disorder of bilirubin metabolism that is caused by the abs
Externí odkaz:
https://doaj.org/article/0cdfa4f8c1e84e47b68f369400bd39cb
Autor:
Jenny A, Greig, Camilo, Breton, Scott N, Ashley, Kelly M, Martins, Cassandra, Gorsuch, Joanna K, Chorazeczewski, Thomas, Furmanak, Melanie K, Smith, Yanqing, Zhu, Peter, Bell, Wendy, Shoop, Hui, Li, Jeff, Smith, Ginger, Tomberlin, Peter, Clark, Thomas W, Mitchell, Elizabeth L, Buza, Hanying, Yan, Derek, Jantz, James M, Wilson
Publikováno v:
Human gene therapy. 33(21-22)
Transthyretin amyloidosis (ATTR) is a progressive and fatal disease caused by transthyretin (TTR) amyloid fibril accumulation in tissues, which disrupts organ function. As the TTR protein is primarily synthesized by the liver, liver transplantation c
Autor:
Jenny A. Greig, Hanying Yan, Thomas Furmanak, Alexa N. Avitto, Caitlin Latshaw, Camilo Breton, Melanie K. Smith, James M. Wilson
Publikováno v:
Mol Ther
Our group previously used adeno-associated viral vectors (AAVs) to express an engineered meganuclease specific for a sequence in the PCSK9 gene (M2PCSK9), a clinical target for treating coronary heart disease. Upon testing this nuclease in non-human
Autor:
Jenny A. Greig, Camilo Breton, Kelly M. Martins, Yanqing Zhu, Zhenning He, John White, Peter Bell, Lili Wang, James M. Wilson
Introductory ParagraphEfforts to improve liver gene therapy have focused on next-generation adeno-associated virus (AAV) vector capsids, transgene delivery, and immunomodulating drugs, such as corticosteroids, to avoid destructive T-cell responses. W
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::58544596b0e55c88705510ccddbb3838
https://doi.org/10.1101/2022.03.24.485675
https://doi.org/10.1101/2022.03.24.485675
Publikováno v:
BMC Genomics, Vol 21, Iss 1, Pp 1-12 (2020)
BMC Genomics
BMC Genomics
Background Identifying nuclease-induced double-stranded breaks in DNA on a genome-wide scale is critical for assessing the safety and efficacy of genome editing therapies. We previously demonstrated that after administering adeno-associated viral (AA