CRISPR-Cas9 In Vivo Gene Editing of KLKB1 for Hereditary Angioedema
CRISPR-Cas9 In Vivo Gene Editing of KLKB1 for Hereditary Angioedema
About this item
Full title
Author / Creator
Longhurst, Hilary J. , Lindsay, Karen , Petersen, Remy S. , Fijen, Lauré M. , Gurugama, Padmalal , Maag, David , Butler, James S. , Shah, Mrinal Y. , Golden, Adele , Xu, Yuanxin , Boiselle, Carri , Vogel, Joseph D. , Abdelhady, Ahmed M. , Maitland, Michael L. , McKee, Mark D. , Seitzer, Jessica , Han, Bo W. , Soukamneuth, Samantha , Leonard, John , Sepp-Lorenzino, Laura , Clark, Eliana D. , Lebwohl, David and Cohn, Danny M.
Publisher
United States: Massachusetts Medical Society
Journal title
Language
English
Formats
Publication information
Publisher
United States: Massachusetts Medical Society
Subjects
More information
Scope and Contents
Contents
Hereditary angioedema is a rare genetic disease that leads to severe and unpredictable swelling attacks. NTLA-2002 is an in vivo gene-editing therapy based on clustered regularly interspaced short palindromic repeats (CRISPR)-CRISPR-associated protein 9. NTLA-2002 targets the gene encoding kallikrein B1 (
), with the goal of lifelong control of...
Alternative Titles
Full title
CRISPR-Cas9 In Vivo Gene Editing of KLKB1 for Hereditary Angioedema
Authors, Artists and Contributors
Author / Creator
Lindsay, Karen
Petersen, Remy S.
Fijen, Lauré M.
Gurugama, Padmalal
Maag, David
Butler, James S.
Shah, Mrinal Y.
Golden, Adele
Xu, Yuanxin
Boiselle, Carri
Vogel, Joseph D.
Abdelhady, Ahmed M.
Maitland, Michael L.
McKee, Mark D.
Seitzer, Jessica
Han, Bo W.
Soukamneuth, Samantha
Leonard, John
Sepp-Lorenzino, Laura
Clark, Eliana D.
Lebwohl, David
Cohn, Danny M.
Identifiers
Primary Identifiers
Record Identifier
TN_cdi_proquest_miscellaneous_2921114840
Permalink
https://devfeature-collection.sl.nsw.gov.au/record/TN_cdi_proquest_miscellaneous_2921114840
Other Identifiers
ISSN
0028-4793
E-ISSN
1533-4406
DOI
10.1056/NEJMoa2309149
