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Optimization of scarless human stem cell genome editing
(Oxford University Press, 2013)
Efficient strategies for precise genome editing in human-induced pluripotent cells (hiPSCs) will enable sophisticated genome engineering for research and clinical purposes. The development of programmable sequence-specific ...
CAS9 transcriptional activators for target specificity screening and paired nickases for cooperative genome engineering
(2013)
Prokaryotic type II CRISPR-Cas systems can be adapted to enable targeted genome modifications across a range of eukaryotes.1–7. Here we engineer this system to enable RNA-guided genome regulation in human cells by tethering ...
Iterative capped assembly: rapid and scalable synthesis of repeat-module DNA such as TAL effectors from individual monomers
(Oxford University Press, 2012)
DNA built from modular repeats presents a challenge for gene synthesis. We present a solid surface-based sequential ligation approach, which we refer to as iterative capped assembly (ICA), that adds DNA repeat monomers ...
Modeling the mitochondrial cardiomyopathy of Barth syndrome with iPSC and heart-on-chip technologies
(2014)
Studying monogenic mitochondrial cardiomyopathies may yield insights into mitochondrial roles in cardiac development and disease. Here, we combine patient-derived and genetically engineered iPSCs with tissue engineering ...
Genome-wide inactivation of porcine endogenous retroviruses (PERVs)
(American Association for the Advancement of Science (AAAS), 2015)
The shortage of organs for transplantation is a major barrier to the treatment of organ failure. While porcine organs are considered promising, their use has been checked by concerns about transmission of porcine endogenous ...
Targeted and genome-wide sequencing reveal single nucleotide variations impacting specificity of Cas9 in human stem cells
(Nature Pub. Group, 2014)
CRISPR/Cas9 has demonstrated a high-efficiency in site-specific gene targeting. However, potential off-target effects of the Cas9 nuclease represent a major safety concern for any therapeutic application. Here, we knock ...
Titin mutations in iPS cells define sarcomere insufficiency as a cause of dilated cardiomyopathy
(American Association for the Advancement of Science (AAAS), 2015)
Human mutations that truncate the massive sarcomere protein titin [TTN-truncating variants (TTNtvs)] are the most common genetic cause for dilated cardiomyopathy (DCM), a major cause of heart failure and premature death. ...