680 research outputs found

    GDNF and p75 Neurotrophin Receptor in Development and Disease

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    Elinkeinorakenteen muutoksista Suomessa

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    Vesistöjen veden laadun muutoksista 1960-luvulla

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    Engl. summary: Changes of water quality in water courses in the 1960'

    Tutkijan motivaatio

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    Teema : motivaati

    Curative gene therapies for rare diseases

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    Diseases caused by alterations in the DNA can be overcome by providing the cells or tissues with a functional copy of the mutated gene. The most common form of gene therapy implies adding an extra genetic unit into the cell. However, new genome engineering techniques also allow the modification or correction of the existing allele, providing new possibilities, especially for dominant diseases. Gene therapies have been tested for 30 years in thousands of clinical trials, but presently, we have only three authorised gene therapy products for the treatment of inherited diseases in European Union. Here, we describe the gene therapy alternatives already on the market in the European Union and expand the scope to some clinical trials. Additionally, we discuss the ethical and regulatory issues raised by the development of these new kinds of therapies.Peer reviewe

    PlasmaDNA: a free, cross-platform plasmid manipulation program for molecular biology laboratories

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    <p>Abstract</p> <p>Background</p> <p>Most molecular biology experiments, and the techniques associated with this field of study, involve a great deal of engineering in the form of molecular cloning. Like all forms of engineering, perfect information about the starting material is crucial for successful completion of design and strategies.</p> <p>Results</p> <p>We have generated a program that allows complete <it>in silico </it>simulation of the cloning experiment. Starting with a primary DNA sequence, PlasmaDNA looks for restriction sites, open reading frames, primer annealing sequences, and various common domains. The databases are easily expandable by the user to fit his most common cloning needs. PlasmaDNA can manage and graphically represent multiple sequences at the same time, and keeps in memory the overhangs at the end of the sequences if any. This means that it is possible to virtually digest fragments, to add the digestion products to the project, and to ligate together fragments with compatible ends to generate the new sequences. Polymerase Chain Reaction (PCR) fragments can also be virtually generated using the primer database, automatically adding to the fragments any 5' extra sequences present in the primers.</p> <p>Conclusion</p> <p>PlasmaDNA is a program available both on Windows and Apple operating systems, designed to facilitate molecular cloning experiments by building a visual map of the DNA. It then allows the complete planning and simulation of the cloning experiment. It also automatically updates the new sequences generated in the process, which is an important help in practice. The capacity to maintain multiple sequences in the same file can also be used to archive the various steps and strategies involved in the cloning of each construct. The program is freely available for download without charge or restriction.</p

    Uuden ihmisen syntymä

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    Teema : syntym

    Kun valo syttyy, olen olemassa

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