901,199 research outputs found

    Translational and Regulatory Challenges for Exon Skipping Therapies

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    Several translational challenges are currently impeding the therapeutic development of antisense-mediated exon skipping approaches for rare diseases. Some of these are inherent to developing therapies for rare diseases, such as small patient numbers and limited information on natural history and interpretation of appropriate clinical outcome measures. Others are inherent to the antisense oligonucleotide (AON)-mediated exon skipping approach, which employs small modified DNA or RNA molecules to manipulate the splicing process. This is a new approach and only limited information is available on long-term safety and toxicity for most AON chemistries. Furthermore, AONs often act in a mutation-specific manner, in which case multiple AONs have to be developed for a single disease. A workshop focusing on preclinical development, trial design, outcome measures, and different forms of marketing authorization was organized by the regulatory models and biochemical outcome measures working groups of Cooperation of Science and Technology Action: "Networking towards clinical application of antisense-mediated exon skipping for rare diseases." The workshop included participants from patient organizations, academia, and members of staff from the European Medicine Agency and Medicine Evaluation Board (the Netherlands). This statement article contains the key outcomes of this meeting.status: publishe

    Detection of Polyps via Shape and Appearance Modeling

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    Presented at the MICCAI 2008 Workshop on Computational and Visualization Challenges in the New Era of Virtual Colonoscopy, September 6, 2008, New York, USA.This paper describes a CAD system for the detection of colorectal polyps in CT. It is based on stochastic shape and appearance modeling of structures of the colon and rectum, in contrast to the data-driven approaches more commonly found in the literature it derives predictive stochastic models for the features used for classification. The method makes extensive use of medical domain knowledge in the design of the models and in the setting of their parameters. The proposed approach was successfully tested on challenging datasets acquired under a protocol with little colonic preparation; such protocol reduces patient discomfort and potentially improves compliance

    OntoMaven: Maven-based Ontology Development and Management of Distributed Ontology Repositories

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    In collaborative agile ontology development projects support for modular reuse of ontologies from large existing remote repositories, ontology project life cycle management, and transitive dependency management are important needs. The Apache Maven approach has proven its success in distributed collaborative Software Engineering by its widespread adoption. The contribution of this paper is a new design artifact called OntoMaven. OntoMaven adopts the Maven-based development methodology and adapts its concepts to knowledge engineering for Maven-based ontology development and management of ontology artifacts in distributed ontology repositories.Comment: Pre-print submission to 9th International Workshop on Semantic Web Enabled Software Engineering (SWESE2013). Berlin, Germany, December 2-5, 201

    Designing experiences with wearables: A case study exploring the blurring boundaries of art, design, technology, culture and distance

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    This paper details a workshop aimed at exploring opportunities for experience design through wearable art and design concepts. Specifically it explores the structure of the workshop with respect to facilitating learning through technology in the development of experiential wearable art and design. A case study titled Cloud Workshop: Wearables and Wellbeing; Enriching connections between citizens in the Asia-Pacific region was initiated through a cooperative partnership between Hong Kong Baptist University (HKBU), Queensland University of Technology (QUT) and Griffith University (GU). Digital technologies facilitated collaboration through an inter-disciplinary, inter-national and inter- cultural approach (Facer & Sandford, 2010) between Australia and Hong Kong. Students cooperated throughout a two-week period to develop innovative wearable concepts blending art, design and technology. An unpacking of the approach, pedagogical underpinning and final outcomes revealed distinct educational benefits as well as certain learning and technological challenges of the program. Qualitative feedback uncovered additional successes with respect to student engagement and enthusiasm, while uncovering shortcomings in the delivery and management of information and difficulties with cultural interactions. Potential future versions of the program aim to take advantage of the positives and overcome the limitations of the current pedagogical approach. It is hoped the case study will become a catalyst for future workshops that blur the boundaries of art, design and technology to uncover further benefits and potentials for new outcomes in experience design

    Design Factory New Zealand: A co-creation space where students work in multidisciplinary teams with industry partners to solve complex problems

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    This workshop provided an opportunity for participants to work with Design Factory New Zealand (DFNZ) principles to experience learning as a multidisciplinary team, working on a complex problem. Participants had a chance to see how DFNZ acts as a transformation agent within students, staff, institution and the wider community. DFNZ as a curriculum allows students to explore new themes that challenge the usual paradigms. We encourage students to take ownership of their learning, to be open minded, and to have the freedom to respond to solutions without the shackles of a traditional design process driven by cost. By partnering with industry and exposing students to create solutions for real world problems, DFNZ has the aim of producing global citizens who have a better chance of succeeding in the workplaces of the future. Currently DFNZ has facilitated learning to students from Civil and Mechanical Engineering, Communication, Information Technology, Business, Design and Sports Science. Industry partners working with the DFNZ team tap into a pool of carefully selected and motivated students who are supported to come up with innovative and holistic solutions to their problems. Industry can use DFNZ as an opportunity to solve specific and existing needs of the company, or utilise fresh thinking to approach complex and wider issues. Involvement with the Design Factory can provide industry with critical strategic insight

    Learning by gaming:ANT and critical making

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    Relationships among theory, gaming, learning and socio-technical design are explored in the two contributions which compose the section. The theory in question is ANT, re-interpreted through critical making - an umbrella term for various distinctive practices that link traditional scholarship in the humanities and social sciences to forms of material engagement. Sergio Minniti describes an ongoing project called Game of ANT, which draws upon the critical making approach to design an interactive technology and a workshop experience through which scholars and students can conceptually-materially engage with ANT, hence exploring and approaching it from novel points of view. Game of ANT adopts the Latourian vision of technoscience as war and physically embodies this idea by proposing a sort of war game during which participants play the roles of human or non-human actors engaging with the competitive dynamics of socio-technical life. The commentary by Stefano De Paoli proposes new directions to develop the project, by deepening the concept of game and its value for design and learning processes.</p
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