Zobrazeno 1 - 10
of 36
pro vyhledávání: '"Aamir Mir"'
Autor:
Raed Ibraheim, Phillip W. L. Tai, Aamir Mir, Nida Javeed, Jiaming Wang, Tomás C. Rodríguez, Suk Namkung, Samantha Nelson, Eraj Shafiq Khokhar, Esther Mintzer, Stacy Maitland, Zexiang Chen, Yueying Cao, Emmanouela Tsagkaraki, Scot A. Wolfe, Dan Wang, Athma A. Pai, Wen Xue, Guangping Gao, Erik J. Sontheimer
Publikováno v:
Nature Communications, Vol 12, Iss 1, Pp 1-17 (2021)
Long-term expression of Cas9 following precision genome editing in vivo may lead to undesirable consequences. Here we show that a single-vector, self-inactivating AAV system containing Cas9 nuclease, guide, and DNA donor can use homology-directed rep
Externí odkaz:
https://doaj.org/article/81e517a4261d442a884395258d491bfe
Autor:
Pengpeng Liu, Shun-Qing Liang, Chunwei Zheng, Esther Mintzer, Yan G. Zhao, Karthikeyan Ponnienselvan, Aamir Mir, Erik J. Sontheimer, Guangping Gao, Terence R. Flotte, Scot A. Wolfe, Wen Xue
Publikováno v:
Nature Communications, Vol 12, Iss 1, Pp 1-13 (2021)
Prime editors use a template sequence within their pegRNA to facilitate nucleotide substitutions or local indels. Here the authors use AAVs to deliver a split-intein prime editor in vivo to correct a pathogenic mutation.
Externí odkaz:
https://doaj.org/article/f79d7d69ebf14bc9b101910fdc770c8c
Autor:
Krishna S Ghanta, Zexiang Chen, Aamir Mir, Gregoriy A Dokshin, Pranathi M Krishnamurthy, Yeonsoo Yoon, Judith Gallant, Ping Xu, Xiao-Ou Zhang, Ahmet Rasit Ozturk, Masahiro Shin, Feston Idrizi, Pengpeng Liu, Hassan Gneid, Alireza Edraki, Nathan D Lawson, Jaime A Rivera-Pérez, Erik J Sontheimer, Jonathan K Watts, Craig C Mello
Publikováno v:
eLife, Vol 10 (2021)
Nuclease-directed genome editing is a powerful tool for investigating physiology and has great promise as a therapeutic approach to correct mutations that cause disease. In its most precise form, genome editing can use cellular homology-directed repa
Externí odkaz:
https://doaj.org/article/a4d2a8d974e94e189ee441394236bb61
Autor:
Nadia Amrani, Xin D. Gao, Pengpeng Liu, Alireza Edraki, Aamir Mir, Raed Ibraheim, Ankit Gupta, Kanae E. Sasaki, Tong Wu, Paul D. Donohoue, Alexander H. Settle, Alexandra M. Lied, Kyle McGovern, Chris K. Fuller, Peter Cameron, Thomas G. Fazzio, Lihua Julie Zhu, Scot A. Wolfe, Erik J. Sontheimer
Publikováno v:
Genome Biology, Vol 19, Iss 1, Pp 1-25 (2018)
Abstract Background The development of CRISPR genome editing has transformed biomedical research. Most applications reported thus far rely upon the Cas9 protein from Streptococcus pyogenes SF370 (SpyCas9). With many RNA guides, wildtype SpyCas9 can i
Externí odkaz:
https://doaj.org/article/d001df012bc241759e81c0e2bc14037f
All-in-one adeno-associated virus delivery and genome editing by Neisseria meningitidis Cas9 in vivo
Publikováno v:
Genome Biology, Vol 19, Iss 1, Pp 1-11 (2018)
Abstract Background Clustered, regularly interspaced, short palindromic repeats (CRISPR) and CRISPR-associated proteins (Cas) have recently opened a new avenue for gene therapy. Cas9 nuclease guided by a single-guide RNA (sgRNA) has been extensively
Externí odkaz:
https://doaj.org/article/6ab1316dcca84075aa5b30d20fb4f776
Autor:
Aamir Mir, Julia F. Alterman, Matthew R. Hassler, Alexandre J. Debacker, Edward Hudgens, Dimas Echeverria, Michael H. Brodsky, Anastasia Khvorova, Jonathan K. Watts, Erik J. Sontheimer
Publikováno v:
Nature Communications, Vol 9, Iss 1, Pp 1-9 (2018)
Resistance of gRNA to ubiquitous ribonucleases is required for CRISPR-Cas9-based therapeutics. Here, the authors explore chemical modifications at all positions of the crRNA guide and tracrRNA cofactor, and identify modified versions that are more po
Externí odkaz:
https://doaj.org/article/b945b146d35544ef9d5a4a30d633c3c0
Autor:
Bianca Garcia, Jooyoung Lee, Alireza Edraki, Yurima Hidalgo-Reyes, Steven Erwood, Aamir Mir, Chantel N. Trost, Uri Seroussi, Sabrina Y. Stanley, Ronald D. Cohn, Julie M. Claycomb, Erik J. Sontheimer, Karen L. Maxwell, Alan R. Davidson
Publikováno v:
Cell Reports, Vol 29, Iss 7, Pp 1739-1746.e5 (2019)
Summary: CRISPR-Cas9 systems provide powerful tools for genome editing. However, optimal employment of this technology will require control of Cas9 activity so that the timing, tissue specificity, and accuracy of editing may be precisely modulated. A
Externí odkaz:
https://doaj.org/article/1d87a0cca2244009993f71c2da8de9ea
Autor:
Jooyoung Lee, Aamir Mir, Alireza Edraki, Bianca Garcia, Nadia Amrani, Hannah E. Lou, Ildar Gainetdinov, April Pawluk, Raed Ibraheim, Xin D. Gao, Pengpeng Liu, Alan R. Davidson, Karen L. Maxwell, Erik J. Sontheimer
Publikováno v:
mBio, Vol 9, Iss 6 (2018)
ABSTRACT In their natural settings, CRISPR-Cas systems play crucial roles in bacterial and archaeal adaptive immunity to protect against phages and other mobile genetic elements, and they are also widely used as genome engineering technologies. Previ
Externí odkaz:
https://doaj.org/article/235e55b82aeb4666b874a7f3a5f87dce
Autor:
Sukanya Iyer, Aamir Mir, Joel Vega-Badillo, Benjamin P. Roscoe, Raed Ibraheim, Lihua Julie Zhu, Jooyoung Lee, Pengpeng Liu, Kevin Luk, Esther Mintzer, Dongsheng Guo, Josias Soares de Brito, Charles P. Emerson, Phillip D. Zamore, Erik J. Sontheimer, Scot A. Wolfe
Publikováno v:
The CRISPR Journal. 5:685-701
While genome editing has been revolutionized by the advent of CRISPR-based nucleases, difficulties in achieving efficient, nuclease-mediated, homology-directed repair (HDR) still limit many applications. Commonly used DNA donors such as plasmids suff
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::2676939cee7f1aebba005624d2b76eb9
https://doi.org/10.1089/crispr.2022.0058
https://doi.org/10.1089/crispr.2022.0058
Autor:
Nida Javeed, Yueying Cao, Esther Mintzer, Samantha J. Nelson, Zexiang Chen, Athma A. Pai, Phillip W. L. Tai, Suk Namkung, Scot A. Wolfe, Aamir Mir, Erik J. Sontheimer, Stacy Maitland, Eraj Khokhar, Dan Wang, Jiaming Wang, Raed Ibraheim, Guangping Gao, Wen Xue, Tomás Rodríguez, Emmanouela Tsagkaraki
Publikováno v:
Nature Communications, Vol 12, Iss 1, Pp 1-17 (2021)
Nature Communications
Nature Communications
Adeno-associated virus (AAV) vectors are important delivery platforms for therapeutic genome editing but are severely constrained by cargo limits. Simultaneous delivery of multiple vectors can limit dose and efficacy and increase safety risks. Here,
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::0b95aee4d8710ab6bb58a155aa6e53c7
https://doi.org/10.1038/s41467-021-26518-y
https://doi.org/10.1038/s41467-021-26518-y