In utero CRISPR-mediated therapeutic editing of metabolic genes
In utero CRISPR-mediated therapeutic editing of metabolic genes
About this item
Full title
Author / Creator
Rossidis, Avery C. , Stratigis, John D. , Chadwick, Alexandra C. , Hartman, Heather A. , Ahn, Nicholas J. , Li, Haiying , Singh, Kshitiz , Coons, Barbara E. , Li, Li , Lv, Wenjian , Zoltick, Philip W. , Alapati, Deepthi , Zacharias, William , Jain, Rajan , Morrisey, Edward E. , Musunuru, Kiran and Peranteau, William H.
Publisher
New York: Nature Publishing Group US
Journal title
Language
English
Formats
Publication information
Publisher
New York: Nature Publishing Group US
Subjects
More information
Scope and Contents
Contents
In utero gene editing has the potential to prenatally treat genetic diseases that result in significant morbidity and mortality before or shortly after birth. We assessed the viral vector–mediated delivery of CRISPR–Cas9 or base editor 3 in utero, seeking therapeutic modification of
Pcsk9
or
Hpd
in wild-type mice or the murine model of...
Alternative Titles
Full title
In utero CRISPR-mediated therapeutic editing of metabolic genes
Authors, Artists and Contributors
Author / Creator
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Primary Identifiers
Record Identifier
TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_6249685
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_pubmedcentral_primary_oai_pubmedcentral_nih_gov_6249685
Other Identifiers
ISSN
1078-8956
E-ISSN
1546-170X
DOI
10.1038/s41591-018-0184-6
