Browsing by Author "Yang, Luhan"

Now showing items 1-8 of 8

    • CAS9 transcriptional activators for target specificity screening and paired nickases for cooperative genome engineering 

      Mali, Prashant; Aach, John; Stranges, P. Benjamin; Esvelt, Kevin M.; Moosburner, Mark; Kosuri, Sriram; Yang, Luhan; Church, George M. (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 ...

    • Development of Human Genome Editing Tools for the Study of Genetic Variations and Gene Therapies 

      Yang, Luhan (2013-10-17)
      The human genome encodes information that instructs human development, physiology, medicine, and evolution. Massive amount of genomic data has generated an ever-growing pool of hypothesis. Genome editing, broadly defined ...

    • Genome-wide inactivation of porcine endogenous retroviruses (PERVs) 

      Yang, Luhan; Guell, Marc; Niu, D.; George, H.; Lesha, E.; Grishin, Dennis; Aach, John Dennis; Shrock, Ellen Lee; Xu, W.; Poci, Jurgen; Cortazio, R.; Wilkinson, R. A.; Fishman, Jay Alan; Church, George McDonald (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 ...

    • Iterative capped assembly: rapid and scalable synthesis of repeat-module DNA such as TAL effectors from individual monomers 

      Briggs, Adrian; Rios, Xavier; Chari, Rajagopal; Yang, Luhan; Zhang, Feng; Mali, Prashant; Church, George McDonald (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 

      Wang, Gang; McCain, Megan L.; Yang, Luhan; He, Aibin; Pasqualini, Francesco Silvio; Agarwal, Ashutosh; Yuan, Hongyan; Jiang, Dawei; Zhang, Donghui; Zangi, Lior; Geva, Judith; Roberts, Amy E.; Ma, Qing; Ding, Jian; Chen, Jinghai; Wang, Da-zhi; Li, Kai; Wang, Jiwu; Wanders, Ronald J. A.; Kulik, Wim; Vaz, Frédéric M.; Laflamme, Michael A.; Murry, Charles E.; Chien, Kenneth R.; Kelley, Richard I.; Church, George M.; Parker, Kevin Kit; Pu, William T. (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 ...

    • Optimization of scarless human stem cell genome editing 

      Yang, Luhan; Guell, Marc; Byrne, Susan; Yang, Joyce L.; De Los Angeles, Alejandro; Mali, Prashant; Aach, John; Kim-Kiselak, Caroline; Briggs, Adrian W; Rios, Xavier; Huang, Po-Yi; Daley, George; Church, George (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 ...

    • Targeted and genome-wide sequencing reveal single nucleotide variations impacting specificity of Cas9 in human stem cells 

      Yang, Luhan; Grishin, Dennis; Wang, Gang; Aach, John; Zhang, Cheng-Zhong; Chari, Raj; Homsy, Jason; Cai, Xuyu; Zhao, Yue; Fan, Jian-Bing; Seidman, Christine; Seidman, Jonathan; Pu, William; Church, George (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 

      Hinson, John Travis; Chopra, Anant; Nafissi, N.; Polacheck, William; Benson, Craig Carlyle; Swist, S.; Gorham, Joshua McClean; Yang, Luhan; Schafer, S.; Sheng, Calvin Chen; Haghighi, Alireza; Homsy, Jason George; Hubner, N.; Church, George McDonald; Cook, S. A.; Linke, Wolfgang; Chen, Christopher; Seidman, Jonathan G.; Seidman, Christine Edry (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. ...