Zobrazeno 1 - 4
of 4
pro vyhledávání: '"Samar Z Rizvi"'
Targeted genome editing in vivo corrects a Dmd duplication restoring wild‐type dystrophin expression
Autor:
Eleonora Maino, Daria Wojtal, Sonia L Evagelou, Aiman Farheen, Tatianna W Y Wong, Kyle Lindsay, Ori Scott, Samar Z Rizvi, Elzbieta Hyatt, Matthew Rok, Shagana Visuvanathan, Amanda Chiodo, Michelle Schneeweiss, Evgueni A Ivakine, Ronald D Cohn
Publikováno v:
EMBO Molecular Medicine, Vol 13, Iss 5, Pp 1-15 (2021)
Abstract Tandem duplication mutations are increasingly found to be the direct cause of many rare heritable diseases, accounting for up to 10% of cases. Unfortunately, animal models recapitulating such mutations are scarce, limiting our ability to stu
Externí odkaz:
https://doaj.org/article/79b8670d90524c9f9b3d247d4ec1653b
Autor:
Eleonora Maino, Ori Scott, Samar Z. Rizvi, Shagana Visuvanathan, Youssif Ben Zablah, Hongbin Li, Ameet S. Sengar, Michael W. Salter, Zhengping Jia, Janet Rossant, Ronald D. Cohn, Bin Gu, Evgueni A. Ivakine
MECP2 duplication syndrome (MDS) is a neurodevelopmental disorder caused by tandem duplication of theMECP2locus and its surrounding genes, includingIRAK1. Current MDS mouse models involve transgenic expression ofMECP2only, limiting their applicabilit
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::a01d3f8950a8e3cccd379007546a6c4c
https://doi.org/10.1101/2023.02.07.527511
https://doi.org/10.1101/2023.02.07.527511
Autor:
Samar Z. Rizvi, Wing Suen Chan, Eleonora Maino, Sydney Steiman, Georgiana Forguson, Maya Klepfish, Ronald D. Cohn, Evgueni A. Ivakine
Publikováno v:
Molecular Therapy: Nucleic Acids, Vol 35, Iss 4, Pp 102356- (2024)
Recent progress in genome editing technologies has catalyzed the generation of sophisticated cell models; however, the precise modeling of copy-number variation (CNV) diseases remains a significant challenge despite their substantial prevalence in th
Externí odkaz:
https://doaj.org/article/16631687f3624b21a3da29b4dc879eca
Autor:
Eleonora Maino, Ori Scott, Samar Z. Rizvi, Wing Suen Chan, Shagana Visuvanathan, Youssif Ben Zablah, Hongbin Li, Ameet S. Sengar, Michael W. Salter, Zhengping Jia, Janet Rossant, Ronald D. Cohn, Bin Gu, Evgueni A. Ivakine
Publikováno v:
Disease Models & Mechanisms, Vol 17, Iss 7 (2024)
Externí odkaz:
https://doaj.org/article/102714651e424d538637bc0d58cdac43