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Pinar Akcakaya
AstraZeneca
4Publications
3H-index
66Citations
Publications 4
Newest
#1Alba Carreras (Sahlgrenska University Hospital)H-Index: 4
#2Luna Simona PaneH-Index: 2
Last.Marcello MarescaH-Index: 6
view all 20 authors...
Background Plasma concentration of low-density lipoprotein (LDL) cholesterol is a well-established risk factor for cardiovascular disease. Inhibition of proprotein convertase subtilisin/kexin type 9 (PCSK9), which regulates cholesterol homeostasis, has recently emerged as an approach to reduce cholesterol levels. The development of humanized animal models is an important step to validate and study human drug targets, and use of genome and base editing has been proposed as a mean to target diseas...
1 CitationsSource
#1Beeke Wienert (University of California, Berkeley)H-Index: 8
#2Stacia K. Wyman (University of California, Berkeley)H-Index: 4
Last.Jacob E. Corn (University of California, Berkeley)H-Index: 32
view all 14 authors...
CRISPR-Cas genome editing induces targeted DNA damage but can also affect off-target sites. Current off-target discovery methods work using purified DNA or specific cellular models but are incapable of direct detection in vivo. We developed DISCOVER-Seq (discovery of in situ Cas off-targets and verification by sequencing), a universally applicable approach for unbiased off-target identification that leverages the recruitment of DNA repair factors in cells and organisms. Tracking the precise recr...
13 CitationsSource
#1Pinar Akcakaya (AstraZeneca)H-Index: 3
#2Maggie L. Bobbin (Harvard University)H-Index: 4
Last.J. Keith Joung (Harvard University)H-Index: 62
view all 22 authors...
CRISPR–Cas genome-editing nucleases hold substantial promise for developing human therapeutic applications1–6 but identifying unwanted off-target mutations is important for clinical translation7. A well-validated method that can reliably identify off-targets in vivo has not been described to date, which means it is currently unclear whether and how frequently these mutations occur. Here we describe ‘verification of in vivo off-targets’ (VIVO), a highly sensitive strategy that can robustly identi...
48 CitationsSource
#1Pinar Akcakaya (AstraZeneca)H-Index: 3
#2Maggie L. Bobbin (Harvard University)H-Index: 1
Last.J. Keith Joung (Harvard University)H-Index: 62
view all 22 authors...
CRISPR-Cas genome-editing nucleases hold substantial promise for human therapeutics but identifying unwanted off-target mutations remains an important requirement for clinical translation. For ex vivo therapeutic applications, previously published cell-based genome-wide methods provide potentially useful strategies to identify and quantify these off-target mutation sites. However, a well-validated method that can reliably identify off-targets in vivo has not been described to date, leaving the q...
4 CitationsSource
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