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Rational design of ...
Rational design of a compact CRISPR-Cas9 activator for AAV-mediated delivery
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- Vora, Suhani (author)
- Massachusetts Institute of Technology
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- Cheng, Jenny (author)
- Harvard University
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Xiao, Ru (author)
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- VanDusen, Nathan J. (author)
- Boston Children's Hospital
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- Quintino, Luis (author)
- Lund University,Lunds universitet,CNS Genterapi,Forskargrupper vid Lunds universitet,CNS Gene Therapy,Lund University Research Groups
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- Pu, William T. (author)
- Boston Children's Hospital
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Vandenberghe, Luk H. (author)
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Chavez, Alejandro (author)
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- Church, George (author)
- Broad Institute
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(creator_code:org_t)
- Cold Spring Harbor Laboratory, 2018
- English.
- Related links:
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http://dx.doi.org/10... (free)
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https://www.biorxiv....
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https://lup.lub.lu.s...
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https://doi.org/10.1...
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Abstract
Subject headings
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- Akin to Zinc Finger and Transcription Activator Like Effector based transcriptional modulators, nuclease-null CRISPR-Cas9 provides a groundbreaking programmable DNA binding platform, begetting an arsenal of targetable regulators for transcriptional and epigenetic perturbation, by either directly tethering, or recruiting, transcription enhancing effectors to either component of the Cas9/guide RNA complex. Application of these programmable regulators is now gaining traction for the modulation of disease-causing genes or activation of therapeutic genes, in vivo. Adeno-Associated Virus (AAV) is an optimal delivery vehicle for in vivo delivery of such regulators to adult somatic tissue, due to the efficacy of viral delivery with minimal concerns about immunogenicity or integration. However, present Cas9 activator systems are notably beyond the packaging capacity of a single AAV delivery vector capsid. Here, we engineer a compact CRISPR-Cas9 activator for convenient AAV-mediated delivery. We validate efficacy of the CRISPR-Cas9 transcriptional activation using AAV delivery in several cell lines.
Subject headings
- NATURVETENSKAP -- Biologi -- Biokemi och molekylärbiologi (hsv//swe)
- NATURAL SCIENCES -- Biological Sciences -- Biochemistry and Molecular Biology (hsv//eng)
Publication and Content Type
- ovr (subject category)
- vet (subject category)
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