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Since 1990 Telethon, along with millions of Italians, has stepped up to the challenge of beating muscular dystrophy and the other genetic diseases. It is a marathon against time, because there are many people who live with these rare disorders, and the resources to deal with them have to be carefully measured out because there is not much public or private funding invested in this field of research, and the path to finding cures is often long and tortuous. The foundation In order to guarantee as much research funding as possible into muscular dystrophy and other genetic diseases, the Telethon team works throughout the year and has adopted a management system for the donated funds that is strict and efficient. For every euro raised by Telethon, about eighty euro cents reach the cutting edge laboratories and excellent research centers. Scientific area The selection of the best research projects, the funding of dedicated researchers and the foundation and maintenance of its research institutes make Telethon a point of Italian excellence in the world. Along with recognition from the international scientific community, Telethon's world of research is the biggest ally of all the people who live with muscular dystrophy or other genetic disorders every day. The online database provides complete information about the projects funded by Telethon from 1991 to the present. The archive contains information about all the Foundation's efforts in the field of biomedical research. In addition to a search by disease, it is possible, using the advanced search function, to interrogate the database by groups of disorders, by the name of a researcher or research institute, or by the town, province, or region where projects are based. The use of another search filter makes it possible to check which research projects are ongoing and which have come to an end.
Proper citation: Telethon Foundation (RRID:SCR_003803) Copy
A project that will study 2,500 people with various systemic autoimmune diseases (SADs), gathering data on the molecular causes of their disease as well as their clinical symptoms, enabling them to pave the way for a new classification of these diseases. The goal is to use OMICs and bioinformatics to identify new classifications for diseases known to share common pathophysiological mechanisms. Results will be shared to deliver a new molecular taxonomy of systemic autoimmune diseases. In order to achieve this, PRECISEADS is informing on a regular basis on the project implementation and results, and involving stakeholders from Europe in its activities in order to broaden the project impact and increase opportunities for cooperation.
Proper citation: PRECISESADS (RRID:SCR_003882) Copy
Consortium to develop efficient and safe strategies for the production of clinical-grade protein pharmaceuticals in plants, and to define the procedures needed for the production of these proteins in compliance with the strict regulatory standards that govern the manufacture of all pharmaceuticals. Ultimately the consortium aimed to take a candidate product all the way through the development pipeline culminating in a phase I human clinical trial. The consortium has a wide range of expertise spanning the areas of molecular biology, plant biology, immunology, recombinant protein expression technology, vaccinology, and plant biotechnology. The objectives listed at the beginning of the Pharma-Planta project are as follows: # To produce a recombinant pharmaceutical molecule in transgenic plants, which will be developed through all regulatory requirements, GMP (good manufacturing practice) standards and pre-clinical toxicity testing. This will then be evaluated in Phase I human clinical trials. # To develop robust risk assessment practices for recombinant pharmaceutical molecules produced in plants, based on health and environmental impact, working with regulatory authorities within the EU as well as public groups to ensure that the production systems are as safe and as acceptable as possible, and that they comply with all biosafety regulations. # To define and carry out a coordinated program for securing and managing intellectual property that will facilitate the availability of high priority plant-derived recombinant pharmaceuticals to the poor in developing countries while simultaneously allowing the products to be developed commercially in Europe and North America. # To develop and refine new strategies for the expression of recombinant pharmaceuticals in plants, which can be used on a generic basis for molecules that are normally expressed poorly. # To develop and generate transgenic plants expressing a second generation of recombinant molecules that will be used in future clinical trials. In 2011 they reached their benchmark for success launching a phase I clinical study of an antibody that neutralizes HIV, produced in and isolated from tobacco plants. This antibody could one day become an inexpensive component of a microbicide used to prevent the spread of HIV/AIDS. The project has also spun off many additional technologies that are being adopted by researchers all over the world, and has resulted in more than 100 publications in peer-reviewed scientific journals.
Proper citation: Pharma-Planta Consortium (RRID:SCR_003880) Copy
Consortium aiming to produce regulatory T cells that are compatible with a kidney transplant patient''''s immune system, as a measure to suppress the body''''s natural immune response against a transplanted organ. If successful, this approach will reduce a transplantation patient''''s life-long dependency on immune suppressing drugs, many of which are often associated with undesirable side effects and can limit the patient''''s daily routine. The consortium goals are to develop and conduct clinical trials of various immunoregulatory T-cell-based products in organ transplantation recipients, allowing a direct comparison of the safety, clinical practicality and therapeutic efficacy of each cell type. The central focus of the project is to: # Production and manufacture of distinct populations of hematopoietic immunoregulatory T cells # Comparatively study the tolerogenic characteristics of these regulatory cell types # Test these cell therapy products side by side in a clinical trial living donor renal transplant recipients The first workstream will work with different T regulatory cell, tolerogenic DC and suppressive macrophage cell products that are currently in development. In addition to these therapeutics, another goal of this workstream is to develop a cell tracking technology that assesses pharmacodynamics and pharmacokinetics of these cell-based therapies. The second workstream is focused on designing and conducting a cell therapy based clinical trial in renal transplantation, taking into consideration ethics, concurrent immunosuppressive drug use, state-of-the-art immune monitoring, innovative all-in-one data capturing systems, and pharmacovigilance. The goal is to have a comparative evaluation of hematopoietic cell therapy safety in renal transplantation. The third workstream aims to learn more about the specific comparative characteristics of suppressive cell types and to use this knowledge to improve later trial designs and foster novel ideas for new or improved suppressive / tolerogenic cell population.
Proper citation: ONE Study (RRID:SCR_003886) Copy
Consortium to accelerate new breakthrough DNA sequencing technologies and methods to enhance existing analysis methods. The ultimate aim is to advance DNA sequencing technologies to a level where a human genome can be analyzed at high resolution for less than 1000 euros in less than one day. The goals of the consortium are to revolutionize nucleic acid analysis methods by improving elements necessary to use the currently emerging generation of nucleic acid sequencers in a meaningful and accessible way, providing methods that allow in situ nucleic acid analysis and methods capable of selectively characterizing mutant DNA in a high background of wildtype DNA, combining RNA and DNA analysis in a single analytical device, providing technology to efficiently analyze DNA methylation (genome-wide, with high resolution and in its long-range context), implementing novel concepts for high-throughput HLA-screening, developing fully integrated solutions for mutational screening of small target regions (such as for screening newborns for cystic fibrosis mutations), developing a device for screening multiple target regions with high accuracy and implementing strategies for effective and high-resolution genotyping of copy number variations. READNA was awarded the Stars of Europe prize in December 2013.
Proper citation: READNA (RRID:SCR_003884) Copy
Project that aims to increase understanding of how orally-administered drugs are taken up from the gastrointestinal tract into the body, and apply this knowledge to create new laboratory tests and computer models that will better predict the performance of these drugs in patients over a range of clinically relevant conditions. The integration of in vitro and in silico approaches will provide a biopharmaceutics toolkit, validated using clinical data, to accelerate drug development. Ultimately, the project will help to facilitate and speed up the formulation development process and significantly reduce the need for animal experiments in this area as well as for human clinical studies in the future. For patients, the main benefit will be in the form of high quality medicines where the dose required is well calculated and is released in a way that consistently provides an optimal clinical effect.
Proper citation: ORBITO (RRID:SCR_003876) Copy
A computationally-oriented biotech company located in Barcelona that is focused on the drug discovery and development areas. Chemotargets' goal is to help the biopharma industry fast-forward the process of bringing new medicines to market, speeding up drug discovery and development programs and making them more cost-efficient. To this end, they have designed, and continue to develop, an integrated technology platform involving a broad spectrum of cutting-edge computational methodologies.
Proper citation: Chemotargets (RRID:SCR_003793) Copy
A non-governmental, non-profit public database for paleontological data providing researchers and the public with information about the entire fossil record. It has been organized and operated by a multi-disciplinary, multi-institutional, international group of paleobiological researchers. Its purpose is to provide global, collection-based occurrence and taxonomic data for organisms of all geological ages, as well data services to allow easy access to data for independent development of analytical tools, visualization software, and applications of all types. The Database's broader goal is to encourage and enable data-driven collaborative efforts that address large-scale paleobiological questions. Paleontological data files are accepted for upload. However, PaleoBioDB needs some basic data types to be included in order to perform an upload. The Application Programming Interface (API) gives scientists, students, and developers programmatic access to taxonomic, spatial, and temporal data contained within the database.
Proper citation: Paleobiology Database (RRID:SCR_003798) Copy
Project targeting vulnerable plaques causing unexpected acute myocardial infarcts and sudden cardiac deaths by identifying and validating novel drug targets as well as devising and validating diagnostic tests. It has been designed to advance the present knowledge on the role of inflammatory remodeling in the different stages of atherosclerosis. It will also provide important knowledge for the development of strategies for prevention and clinical management of vascular diseases.
Proper citation: AtheroRemo (RRID:SCR_003831) Copy
An open-source Java viewer for chemical structures in 3D with features for chemicals, crystals, materials and biomolecules. It is cross-platform, running on Windows, Mac OS X, and Linux/Unix systems and features an applet, application, and systems integration component.
Proper citation: Jmol (RRID:SCR_003796) Copy
https://www.biogen.com/en_us/home.html
Global biotechnology company based in Cambridge, Massachusetts, specializing in discovering, developing, and delivering important therapies for the treatment of neurodegenerative, hematologic and autoimmune diseases to patients worldwide.
Proper citation: Biogen Idec (RRID:SCR_003790) Copy
Organization that drives the development of disease-modifying treatments against Alzheimer's, Parkinson's, Diabetes and other protein misfolding disorders through two independently managed business units: * Contract Research: The in-vivo Contract Research Organization (CRO) helps its clients to assess the pharmacokinetics and -dynamics of their experimental treatments against Alzheimer's disease. The main focus is on efficacy testing of candidate drugs in reMYND's proprietary Alzheimer mouse models expressing the clinical APP-London allele as single transgene or in combination with clinical alleles of human PS1 and TAU. * Drug Discovery: The Drug Discovery and Development Unit (DDD) focuses on disease-modifying treatments against protein-misfolding disorders, such as Alzheimer's disease (tau), Parkinson's disease (-synuclein), and Diabetes. In addition, reMYND grants licenses and markets commercial kits of RadarScreen, a technology for rapid and cost-effective identification of genotoxic liabilities in early stage drug discovery. reMYND has been substantially supported by grants from IWT and from The Michael J Fox Foundation.
Proper citation: reMYND (RRID:SCR_003824) Copy
A bio-pharmaceutical company with a focus on research and development of new targets & therapies against proliferative diseases. Its current development program is based on inecalcitol, a vitamin D receptor agonist being studied in three potential indications: * alone in chronic lymphocytic leukaemia * in combination with imatinib in chronic myeloid leukaemia * in prostate cancer, for use with current standards of care Hybrigenics'' research program investigates the action of enzymes called Deubiquitinating Enzymes (DUBs) in the recycling of onco-proteins and the utility of proprietary patented DUB inhibitors against various cancer indications. Hybrigenics Services, a Hybrigenics subsidiary, markets very specialized scientific services to researchers in all areas of life sciences who want to identify, validate and inhibit protein interactions in animal, plant or microbiological cells.
Proper citation: Hybrigenics (RRID:SCR_003822) Copy
Foldit is a revolutionary new multiplayer online computer game that engages non-scientists in solving hard prediction problems, enabling you to contribute to important scientific research. Foldit players interact with protein structures using direct manipulation tools and user-friendly versions of algorithms from the Rosetta structure prediction methodology, while they compete and collaborate to optimize the computed energy. Here are the basic principles to keep in mind when folding proteins. Your score on each protein is based on how well you do with these three things: # Pack the protein: The smaller the protein, the better. More precisely, you want to avoid empty spaces (voids) in the structure of the protein where water molecules can get inside. So you want the atoms in the protein to be as close together as possible. Certain structures, such as sheets, will even connect together with hydrogen bonds if you line them up right and get them close together. This is also good. Key word: Compact. # Hide the hydrophobics: Hydrophobics are the sidechains that don't want to be touching water, just like oil or wax. Since most proteins float around in water, you want to keep the hydrophobics (orange sidechains) surrounded by as many atoms as possible so the water won't get to them. The other side of this rule is that hydrophilics (blue sidechains) do want to be touching water, so they should be exposed as much as possible. Key word: Buried. # Clear the clashes: Two atoms can't occupy the same space at the same time. If you've folded a protein so two sidechains are too close together, your score will go down a lot. This is represented by a red spiky ball (clash) where the two sidechains are intersecting. If there are clashes, you know something is wrong with your protein. So make sure everything is far enough apart. Key word: Apart. The current series of Science Puzzles, the Grand Challenges, are meant to generate the evidence needed to prove that human protein folders can be more effective than computers at certain aspects of protein structure prediction. That's what all the puzzles in Foldit are about right now: predicting the structure of a protein based on its amino acid sequence. The three rules mentioned above describe the characteristics of correct protein structures.
Proper citation: Foldit (RRID:SCR_003788) Copy
Private research university in Rochester, New York. University grants undergraduate and graduate degrees, including doctoral and professional degrees.
Proper citation: University of Rochester; New York; USA (RRID:SCR_003821) Copy
Product development company focused on therapeutic products and diagnostic devices targeting misfolded protein diseases. On July, 2015 the company name was changed to ProMIS Neurosciences, Inc.
Proper citation: Amorfix (RRID:SCR_003783) Copy
http://www.patientslikeme.com/
A for-profit health data-sharing platform that can transform the way patients manage their conditions, change the way industry conducts research and improve patient care. PatientsLikeMe aligns patient and industry interests through data-sharing partnerships. They work with trusted nonprofit, research and industry Partners who use this health data to improve products, services and care for patients. They take the information patients share about their experience with the disease and sell it to their partners (i.e., companies that are developing or selling products to patients). These products may include drugs, devices, equipment, insurance, and medical services. Except for the restricted personal information entered when registering for the site, participants should expect that every piece of information submitted (even if it is not currently displayed) may be shared with their partners and any member of PatientsLikeMe, including other patients. They do not rent, sell or share personally identifiable information for marketing purposes or without explicit consent. Because they believe in transparency, they tell members exactly what they do and do not do with their data. Patients have the opportunity to share both personal stories and health data about their conditions to help uncover great ideas and new knowledge. By sharing information on the site, they can put their disease experiences in context and find answers to the questions they have. Every partnership we develop must bring them closer to aligning patient and industry interests. Their end goal is improved patient care and quality of life.
Proper citation: PatientsLikeMe (RRID:SCR_003781) Copy
http://www.innomed-addneuromed.com/
THIS RESOURCE IS NO LONGER IN SERVICE. Documented on January 9,2023. Project portal for a cross European study designed to find biomarkers, or tests, for Alzheimer's disease. Its objectives are to produce and improve experimental models of Alzheimer's for biomarker discovery and to identify a biomarker for Alzheimer's disease suitable for diagnosis, prediction, and monitoring disease progression for use in clinical trials and in clinical practice. The baseline dataset database was scheduled to be completed and locked in 2008 and become available to researchers by 2009. Requests to access the data will be reviewed by the scientific projects committee.
Proper citation: AddNeuroMed (RRID:SCR_003819) Copy
Unit studying human cognition and the brain with about 90 researchers and postgraduate students investigating topics such as attention, emotion, language and memory. They are developing new treatments for depression, improving hearing through cochlear implants, and helping children to overcome memory problems. With a large collection of scientists engaged in both basic and translational research on the mind and brain, the Unit provides an exceptional training and academic environment that benefits postgraduate students and researchers at all levels. A significant part of their research makes use of brain imaging and they have excellent on-site facilities for magnetic resonance imaging (MRI) magnetoencephalography (MEG) and electroencephalography (EEG). They also have clinical facilities at Addenbrooke's Hospital. The Unit has close links both with the hospital and with Cambridge University.
Proper citation: MRC Cognition and Brain Sciences Unit (RRID:SCR_003818) Copy
A human writeable and human readable language to express the information required to describe pharmacometric models and tasks using these models. The specification of the MDL offers a standard for coding models and associated objects and defining how to execute tasks. The standards for defining MDL objects are independent of any specific target modelling software. This allows the user to specify the model in a consistent manner and facilitates understanding of the model and associated tasks, regardless of the software used to build the model. New modelling applications may take advantage of the standard without having to re-invent how to describe common modelling processes.
Proper citation: MDL (RRID:SCR_003816) Copy
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