R
Ruth M. Castellanos Rivera
Researcher at University of North Carolina at Chapel Hill
Publications - 14
Citations - 2665
Ruth M. Castellanos Rivera is an academic researcher from University of North Carolina at Chapel Hill. The author has contributed to research in topics: Genome editing & Adeno-associated virus. The author has an hindex of 8, co-authored 13 publications receiving 2083 citations.
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In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy.
Christopher E. Nelson,Chady H. Hakim,David G. Ousterout,Pratiksha I. Thakore,Eirik A. Moreb,Ruth M. Castellanos Rivera,Sarina Madhavan,Xiufang Pan,F. Ann Ran,F. Ann Ran,Winston X. Yan,Winston X. Yan,Winston X. Yan,Aravind Asokan,Feng Zhang,Dongsheng Duan,Charles A. Gersbach +16 more
TL;DR: In this paper, an adeno-associated virus was used to deliver the clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9 system to the mdx mouse model of Duchenne muscular dystrophy (DMD) to remove the mutated exon 23 from the dystrophin gene.
In vivo genome editing improves muscle function in a mouse model of Duchenne muscular dystrophy
Christopher E. Nelson,Chady H. Hakim,David G. Ousterout,Pratiksha I. Thakore,Eirik A. Moreb,Ruth M. Castellanos Rivera,Sarina Madhavan,X. Pan,F. A. Ran,Winston X. Yan,Aravind Asokan,Feng Zhang,Dongsheng Duan,Charles A. Gersbach +13 more
TL;DR: This work establishes CRISPR-Cas9–based genome editing as a potential therapy to treat DMD and partially restored dystrophin protein expression in skeletal and cardiac muscle and improved skeletal muscle function.
Journal ArticleDOI
Aquaporin-4-dependent glymphatic solute transport in the rodent brain.
Humberto Mestre,Lauren M. Hablitz,Anna L.R. Xavier,Weixi Feng,Wenyan Zou,Tinglin Pu,Hiromu Monai,Giridhar Murlidharan,Ruth M. Castellanos Rivera,Matthew J. Simon,Martin M. Pike,Virginia Plá,Ting Du,Benjamin T. Kress,Xiaowen Wang,Benjamin A. Plog,Alexander S. Thrane,Alexander S. Thrane,Iben Lundgaard,Iben Lundgaard,Yoichiro Abe,Masato Yasui,John H. Thomas,Ming Xiao,Hajime Hirase,Aravind Asokan,Aravind Asokan,Jeffrey J. Iliff +27 more
TL;DR: It is concur that CSF influx is higher in wild-type mice than in four different Aqp4 KO lines and in one line that lacks perivascular AQP4 (Snta1 KO), and it is reported that intrastriatal injections suppress glymphatic function.
Journal ArticleDOI
Long-term evaluation of AAV-CRISPR genome editing for Duchenne muscular dystrophy
Christopher E. Nelson,Yaoying Wu,Matthew Gemberling,Matthew L. Oliver,Matthew A. Waller,Joel D. Bohning,Jacqueline N. Robinson-Hamm,Karen Bulaklak,Ruth M. Castellanos Rivera,Joel H. Collier,Aravind Asokan,Charles A. Gersbach +11 more
TL;DR: The potential of AAV-CRISPR for permanent genome corrections and aspects of host response and alternative genome editing outcomes that require further study are shown and highlighted.
Journal ArticleDOI
Structure-guided evolution of antigenically distinct adeno-associated virus variants for immune evasion
Longping V. Tse,Kelli A. Klinc,Victoria J. Madigan,Ruth M. Castellanos Rivera,Lindsey F. Wells,L. Patrick Havlik,J. Kennon Smith,Mavis Agbandje-McKenna,Aravind Asokan +8 more
TL;DR: These proof-of-principle studies demonstrate that synthetic AAV variants can be evolved to evade neutralizing sera from different species—mice, nonhuman primates, and humans—without compromising yield and transduction efficiency or altering tropism.