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We have previously described a means for directing the growth of genetically modified cells in vivo using a derivative of the thrombopoietin receptor, mpl, that is reversibly activated by a drug called a chemical inducer of dimerization (CID).
By contrast, relatively inefficient recombination is essential for experiments in which the goal is to monitor the behavior, morphology, or function of individual genetically modified cells in an environment populated by unmodified neighbors.
To prove a cell autonomous and specific role for Gfi1, we transduced Gfi1−/− LSK cells with retroviruses encoding Gfi1-GFP and GFP, respectively, and cultured these genetically modified cells in SCF and IL7 containing medium.
These substances have been delivered with implantable infusion devices, viral vectors and transplantation of genetically modified cells in experimental models of PD (Cunningham & Su, 2002; Mohapel et al, 2005; Lang et al, 2006; Sadan et al, 2009; Biju et al, 2010; Marks et al, 2010).
The investigators removed skin cells from the patients' upper arms and grew the modified cells in the laboratory, adding the gene for Factor VIII obtained from healthy humans.
But you can only hold the world back so long now that the tech is out there and Chinese scientists have already started to inject human beings with modified cells in a trial study using the CRISPR-Cas9 technology.
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Great progress has been made to develop biodegradable polymeric vectors for non-viral gene delivery in 2D culture, which generally involves isolating and modifying cells in vitro, followed by subsequent transplantation in vivo.
Due to its flexibility viral infection has become a common tool to genetically label or modify cells in vivo.
Function-spacer-lipid (FSL) constructs have been used to modify cells in vitro and in vivo [ 43, 45– 48], as well as enveloped viral particles [ 14].
There is every reason to argue that cell-type-specific epigenome editing systems should be used to modify cells in vivo and/or in vitro to further basic science as well as correct a variety of diseases.
Recent data suggest that we should not focus exclusively on whether BPs target tumour cells directly, but also consider how these potent anti-resorptive agents modify cells in the bone microenvironment that are essential for tumour growth.
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