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      Cell-permeable nanobodies for targeted immunolabelling and antigen manipulation in living cells

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          Progress and problems with the use of viral vectors for gene therapy.

          Clare Thomas, Anja Ehrhardt, Mark Kay (2003)
          Gene therapy has a history of controversy. Encouraging results are starting to emerge from the clinic, but questions are still being asked about the safety of this new molecular medicine. With the development of a leukaemia-like syndrome in two of the small number of patients that have been cured of a disease by gene therapy, it is timely to contemplate how far this technology has come, and how far it still has to go.
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            Reversal of neurological defects in a mouse model of Rett syndrome.

            Jacky Guy, Jian Gan, Jim Selfridge … (2007)
            Rett syndrome is an autism spectrum disorder caused by mosaic expression of mutant copies of the X-linked MECP2 gene in neurons. However, neurons do not die, which suggests that this is not a neurodegenerative disorder. An important question for future therapeutic approaches to this and related disorders concerns phenotypic reversibility. Can viable but defective neurons be repaired, or is the damage done during development without normal MeCP2 irrevocable? Using a mouse model, we demonstrate robust phenotypic reversal, as activation of MeCP2 expression leads to striking loss of advanced neurological symptoms in both immature and mature adult animals.
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              Synthesis of proteins by native chemical ligation.

              S Kent, P E Dawson, T W Muir … (1994)
              A simple technique has been devised that allows the direct synthesis of native backbone proteins of moderate size. Chemoselective reaction of two unprotected peptide segments gives an initial thioester-linked species. Spontaneous rearrangement of this transient intermediate yields a full-length product with a native peptide bond at the ligation site. The utility of native chemical ligation was demonstrated by the one-step preparation of a cytokine containing multiple disulfides. The polypeptide ligation product was folded and oxidized to form the native disulfide-containing protein molecule. Native chemical ligation is an important step toward the general application of chemistry to proteins.
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                Author and article information

                Journal
                Title: Nature Chemistry
                Abbreviated Title: Nature Chem
                Publisher: Springer Nature
                ISSN (Print): 1755-4330
                ISSN (Electronic): 1755-4349
                Publication date Created: July 17 2017
                Publication date (Print): July 17 2017
                Volume:
                Issue:
                Article
                DOI: 10.1038/nchem.2811
                PubMed ID: 28754949
                SO-VID: fcfded9b-19d2-448f-8e6c-dd11526516aa
                Copyright © © 2017
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