Could editing the DNA of embryos with CRISPR help save people who are already alive?

Amid last year’s backlash against the birth of the world’s first genetically edited babies, some experts preached prudence: Editing the genomes of embryos, they argued, could one day “cure” people of diseases before they’re even born. But there is another, less-discussed potential application of editing an embryo: tweaking its DNA to help save someone who is already alive. Take the case of Jessica and Keith, a couple in the Bay Area with a 2 1/2-year-old daughter with Fanconi anemia, a genetic disease that leads to the failure of bone marrow to produce red and white blood cells and carries an increased risk of a number of cancers. The best treatment is a stem cell transplant from a sibling, and Jessica and Keith, who asked that their last name not be used, are now in the process of trying to have another child through IVF who can serve as a donor — what’s known as a savior sibling.

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ATCC

With almost a century of scientific best practices and know-how, ATCC offers an unmatched combination of being the largest and most diverse collection of biological research solutions and a mission-driven, trusted partner that supports and encourages scientific collaboration. At ATCC, we believe that credible leads to incredible. We are dedicated to raising credibility in science by empowering the global scientific community with the credible standards, cutting-edge biological models, and custom solutions that lead to incredible scientific advancements. Visit atcc.org to learn more about our commitment to the future.

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MedTech

Wisconsin biotech companies could play key roles in long-term economic recovery from COVID-19 pandemic

Article | September 22, 2022

Whether it’s called a modern “Manhattan Project” or a medical moon shot, the concept of long-term economic recovery rests on how confident people are they won’t risk serious illness by venturing forth in public again. Wisconsin stands to be a significant part of such an undertaking, whatever it’s called. The shorter-term debate is well under way over the gradual lifting of COVID-19 emergency rules, such as the now-extended “safer-at-home” order in Wisconsin. At least a dozen states, including regional coalitions on the East and West coasts, are exploring next steps as they seek to balance responses to the virus with calls for reopening the economy, at least, in part. Wisconsin’s ability to shape longer-term responses will come from private and public resources, which range from companies engaged in production of diagnostics.

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Research

Advancement in Genomics Accelerating its Penetration into Precision Health

Article | July 11, 2022

Genomics is an interdisciplinary field of biology emphasizing the structure, editing, evolution, function, and mapping of genomes. It is creating deeper inroads across the precision health domain with the increasing introduction of advanced technologies such as quantum simulation, next-generation sequencing (NGS), and precise genome manipulation. As precision health focuses on providing the proper intervention to the right patient at the right time, genomics increasingly finds applications in human and pathogen genome sequencing in clinical and research spaces. Rising Hereditary Diseases Burden Paving the Way for Genomics in Precision Health In the last few years, a significant surge in the prevalence of diseases and ailments such as diabetes, obesity, baldness, and others has been witnessed across the globe. A history of family members with chronic diseases, such as cancer, diabetes, high blood pressure, hearing issues, and heart disease, can sometimes continue into the next generation. Hence, the study of genes is extensively being conducted for predicting health risks and early treatment of these diseases. It also finds use in CRISPR-based diagnostics and the preparation of precision medication for the individual. In addition, ongoing advancements in genomics are making it possible to identify different genetic traits that persuade people to more widespread diseases and health problems. The Emergence of Genomics Improves Disease Understanding Genomics refers to the study of the complete genetic makeup of a cell or organism. Increasing scientific research in the area substantially contributes to increasing knowledge about the human genome and assists in improving the ability to understand disease etiology, risk, diagnosis, treatment, and prevention. On account of these improvements, innovative genomic technologies and tools are being developed to enable better precision health not only for the individual but for various regional populations as well. The Way Forward With growing preference for personalized medicine and an increasing need for more accurate pathogen detection and diagnostics, genomics is gaining huge popularity across the precision health domain. Also, increasing research activities for developing novel high-precision therapeutics and rising importance of gene study in the prevention, diagnosis, and management of infectious and genetic diseases will further pave the way for genomics in the forthcoming years.

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MedTech

Next-Gen Genetics Cancer Therapies Creating Investment Prospects

Article | July 12, 2022

Genetic therapeutics such as genetic engineering and gene therapy are increasingly emerging as one of the most influential and transformed biotechnological solutions around the globe in recent times. These genetic solutions are being assessed across various medical domains, including cancer treatment, neurology, oncology, and ophthalmology. Citing the trend, the genetics industry is estimated to experience a tsunami of approvals, with over 1,000 cell and gene therapy clinical trials currently underway and over 900 companies worldwide focusing on these cutting-edge therapies. Growing Cancer Encourages Advancements in Genetic Technologies With the surging cases of cancers such as leukemias, carcinomas, lymphomas, and others, patients worldwide are increasing their spending on adopting novel therapeutic solutions for non-recurring treatment of the disease, such as gene therapy, genetic engineering, T-cell therapy, and gene editing. As per a study by the Fight Cancer Organization, spending on the treatment of cancer increased to $200.7 billion, and the amount is anticipated to exceed $245 billion by the end of 2030. Growing revenue prospects are encouraging biotechnology and biopharmaceutical companies to develop novel genetic solutions for cancer treatment. For instance, Bristol-Myers Squibb K.K., a Japanese pharmaceutical company, introduced a B-cell maturation antigen (BCMA)-directed chimeric antigen receptor (CAR) T cell immunotherapy, Abecma, for the treatment of relapsed or refractory (R/R) multiple myeloma in 2022. Amid a New Market: Genetics Will Attract Massive Investments Despite several developments and technological advancements, genetics is still considered to be in a nascent stage, providing significant prospects for growth to the companies that are already operating in the domain. Genetics solutions such as gene therapies, gene editing, and T-cell immunotherapy are emerging as highly active treatments across various medical fields, resulting in increasing research and development activities across the domain, drawing significant attention from investors. Given the potential of genetic treatments and the focus on finding new ways to treat cancer and other related diseases, it's easy to understand why companies are investing in the domain. For instance, Pfizer has recently announced an investment of around $800 million to construct development facilities supporting gene therapy manufacturing from initial preclinical research through final commercial-scale production. Due to these advancements, cell and gene therapies are forecast to grow from $4 billion annually to more than $45 billion, exhibiting growth at a 63% CAGR. The Future of Genetics Though there is a significant rise in advancement in genetic technologies and developments, the number of approved genetic treatments remains extremely small. However, with gene transfer and CRISPR solutions emerging as new modalities for cancer treatment, the start-up companies will attract a growing amount and proportion of private and public investments. This is expected present a tremendous opportunity for biopharma and biotechnology investors to help fund and benefit from the medical industry's shift from traditional treatments to cutting-edge genetic therapeutics in the coming years.

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Medical

Nanostructures: Emerging as Effective Carriers for Drug Delivery

Article | July 14, 2022

Natural remedies have been employed in medicine since antiquity. However, a large number of them fail to go past the clinical trial stages. In vivo instability, poor solubility and bioavailability, a lack of target-specific delivery, poor absorption, and side effects of the medication are only a few of the problems caused by the use of large-sized materials in drug administration. Therefore, adopting novel drug delivery systems with targeted medications may be a solution to address these pressing problems. Nanotechnology has received tremendous attention in recent years and has been demonstrated to help blur the boundaries between the biological and physical sciences. With great success, it plays a vital part in enhanced medication formulations, targeted venues, and controlled drug release and delivery. Limitations of Traditional Delivery Trigger the Adoption of Nanoparticles The field of nanotechnology and the creation of drug formulations based on nanoparticles is one that is expanding and showcasing great potential. It has been thoroughly researched in an effort to develop new methods of diagnosis and treatment and to overcome the limitations of several diseases' current therapies. As a result, nanoparticles are being used to improve the therapeutic effectiveness and boost patient adherence to treatment by increasing medication bioavailability, drug accumulation at a particular spot, and reducing drug adverse effects. The nanoparticles could be transformed into intelligent systems housing therapeutic and imaging agents by manipulating their surface properties, size, correct drug load, and release with targeted drug delivery. Nanostructures facilitate the release of combination medications at the prescribed dose since they remain in the blood circulation system for a long time. Therefore, they result in fewer plasma fluctuations with decreased side effects. Due to their nanoscale, these structures can easily enter the tissue system, promote the absorption of drugs by cells, make medication administration more effective, and ensure that the medicine acts at the targeted location. The Way Ahead Nanomedicine and nano-delivery systems are a comparatively new but fast-evolving science in which nanoscale materials are used as diagnostic tools to deliver drug molecules at precisely targeted sites in a controlled manner. It is finding applications for the treatment of diseases such as cardiovascular, neurodegenerative, cancer, ocular, AIDS, and diabetes, among others. With more research and technological advancement, these drug delivery solutions will open up huge opportunities for companies that work with them.

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Spotlight

ATCC

With almost a century of scientific best practices and know-how, ATCC offers an unmatched combination of being the largest and most diverse collection of biological research solutions and a mission-driven, trusted partner that supports and encourages scientific collaboration. At ATCC, we believe that credible leads to incredible. We are dedicated to raising credibility in science by empowering the global scientific community with the credible standards, cutting-edge biological models, and custom solutions that lead to incredible scientific advancements. Visit atcc.org to learn more about our commitment to the future.

Related News

AI, Diagnostics

Genomic Vision Announces launch of FiberSmart

Genomic Vision | March 08, 2023

On March 7, 2023, Genomic Vision, a leading biotech company that develops products and services for the analysis and control of genome changes, launched FiberSmart®, an AI-based technology for automating the quantification and detection of fluorescent signals on combed DNA molecules. Initially available for the analysis of Replication Combing Assays (RCA), Genomic Vision's proprietary method for directly visualizing DNA replication kinetics at the single molecule level. FiberSmart uses advanced AI methods to detect, visualize, and analyze DNA replication kinetics up to 3x more accurately and up to 10x faster than Genomic Vision's existing software solutions. The technology offers a simple and user-friendly interface, facilitating quick analysis of DNA replication signals to deduce essential parameters describing replication kinetics. It is compatible with Genomic Vision's FiberVision® and FiberVision-S® scanners for the RCA assay of the company's proprietary DNA combing technology with various potential applications, including in gene and cell therapy quality control. FiberSmart has been successfully tested and validated by AstraZeneca and the Fritz Lipmann Institute in Germany. Genomic Vision's CEO Aaron Bensimon said, "The launch of FiberSmart® is an important milestone for Genomic Vision as we bring the benefits of powerful AI technology to our users, who can now perform faster and more accurate genomic analysis seamlessly. Our proprietary DNA combing technique has multiple potential applications, particularly in the cell and gene therapy space, where highly accurate genomic analysis is paramount to ensure robust quality standards are met. With the launch of this software we are making it easier for users to exploit the full potential of our proprietary advanced genomic analysis technique." (Source – Business Wire) About Genomic Vision GENOMIC VISION is a leading biotechnology company specializing in the analysis of genome modifications, with a focus on ensuring their quality and safety in genome editing technologies and biomanufacturing processes. It utilizes advanced nanotechnology for DNA analysis to develop cutting-edge diagnostic and drug discovery solutions for cancer and acute diseases at the intersection of genome dynamics and human diseases. The company's approach employs Molecular Combing Technology, a powerful technique that directly visualizes single DNA molecules to detect and quantify changes in the genome landscape and their contribution to pathology.

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Medical

Spotlight Therapeutics Raises $36.5 Million Series B to Advance a Pipeline of Cell-Targeted In Vivo CRISPR Gene Editing Biologics

Spotlight Therapeutics | March 22, 2022

Spotlight Therapeutics, Inc. (“Spotlight”), a biotechnology company applying new insights to develop cell-targeted in vivo CRISPR gene editing biologics, today announced a $36.5M Series B financing to fuel a drive toward the clinic. The financing round was co-led by new investors GordonMD Global Investments and EPIQ Capital Group, with participation from Magnetic Ventures, as well as existing investors GV (formerly Google Ventures) and Emerson Collective and other investors. Craig Gordon, M.D., Founder, CEO and CIO of GordonMD Global Investments, joins the Company’s Board of Directors. Spotlight's proprietary technology platform, TAGE (Targeted Active Gene Editors), is a new class of biologics; highly engineered, modular programmable CRISPR effectors designed to target and edit selected cell types in vivo. This approach circumvents the complexity of packaged viral, viral-like, and nanoparticle delivery systems, opens the door to expanded applications, and holds the promise of increasing patient access. We are excited to help Spotlight advance its pioneering work, which shows promise for cell-targeted delivery of CRISPR effectors in vivo. Spotlight’s TAGE platform could enable significant expansion of CRISPR medicines to a wide range of diseases." Dr. Gordon. This Series B funding is a crucial milestone as we advance our lead first-in-class immuno-oncology (IO) program and progress our pipeline of programs in IO, ophthalmic diseases and hemoglobinopathies,It will enable us to execute our development plan, leveraging Spotlight’s unique cell-targeted in vivo delivery approach, as we aspire to unlock the full potential of gene editing and enable effective one-and-done medicines for patients.” Mary Haak-Frendscho, Ph.D., President and CEO of Spotlight Therapeutics. About Spotlight Therapeutics Established in mid-2018, Spotlight Therapeutics is a privately held biotechnology company advancing a pipeline of cell-targeted in vivo CRISPR gene editing therapies. Spotlight's proprietary technology platform TAGE (Targeted Active Gene Editors) is a new class of biologics, CRISPR effectors engineered for direct delivery in vivo, to achieve cell-selective therapeutic genome editing. Spotlight's pipeline is advancing its modular programmable CRISPR effectors towards clinical studies in immuno-oncology, ophthalmic diseases and hemoglobinopathies. The company is headquartered in Hayward, California.

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Medical

TGen Selects MemVerge to Accelerate Idiopathic Pulmonary Fibrosis Research Discovery with Big Memory Technology

MemVerge | March 21, 2022

MemVerge™, the pioneers of Big Memory software, today announced that TGen, the Translational Genomics Research Institute, an affiliate of City of Hope, has selected MemVerge Memory Machine Big Memory virtualization software to accelerate time to discovery for Idiopathic Pulmonary Fibrosis (IPF), a disease which affects 100,000 people annually in the U.S. Using MemVerge technology, TGen is able to dramatically speed analytical processing by nearly 36% for single-cell RNA sequencing. As a nonprofit medical research institute, TGen researchers process single-cell RNA sequences to characterize cell transcriptomic profiles. The process can take up to six and a half hours to analyze a matrix of 30,000 genes by 114,000 cells. With consistently growing datasets, this processing time was preventing a desired time to discovery. The data required for analysis was simply too large to retain in traditional memory, and scaling capacity with dynamic random-access memory (DRAM) was too costly. TGen has instead deployed memory virtualization technology from MemVerge which virtualizes both DRAM and PMem (persistent memory) memory technologies, to increase the memory pool available for processing without requiring more high-cost DRAM. The solution further speeds TGen's genomics sequencing analysis with Memory Machine ZeroIO in-memory snapshots which capture multi-terabyte data sets at any point for rapid reloads at each stage of processing. The ZeroIO snapshot service is 1,000 times faster than the fastest storage snapshot to SSD and enables TGen to run processing workflows in parallel. This ensures that in the event of a system crash, in-memory snapshots are available to instantly re-start long running jobs without lengthy reloading. By utilizing the snapshotting and cloning capabilities of Memory Machine, we were able to parallelize the processing workflow, As a result, we can now save nearly 36% of computational time while also taking advantage of the big memory nodes. This will save a lot of time in downstream analysis." Glen Otero, Ph.D., Vice President of Scientific Computing at TGen. MemVerge Memory Machine has quickly resulted in research value for TGen, We have removed performance barriers from their research process so that they are able to perform vital, life-saving, research faster than ever possible. Now TGen is expanding the use of Big Memory technology across other research use cases where results and discoveries can produce findings for a healthier tomorrow." Jonathan Jiang, COO of MemVerge. MemVerge Memory Machine makes 100% use of available memory capacity while providing new operational capabilities to memory-centric workloads. Memory Machine answers the need for a modern in-memory computing model to support emerging applications that require real-time analytics, true in-memory computing, and fault-tolerant memory persistence to speed massive processing workloads. About MemVerge MemVerge is pioneering Big Memory Computing and Big Memory Cloud technology for the memory-centric and multi-cloud future. MemVerge® Memory Machine™ is the industry's first software to virtualize memory hardware for fine-grained provisioning of capacity, performance, availability, and mobility. On top of the transparent memory service, Memory Machine provides another industry first, ZeroIO™ in-memory snapshots which can encapsulate terabytes of application state within seconds and enable data management at the speed of memory. The breakthrough capabilities of Big Memory Computing and Big Memory Cloud Technology are opening the door to cloud agility and flexibility for thousands of Big Memory applications.

Read More

AI, Diagnostics

Genomic Vision Announces launch of FiberSmart

Genomic Vision | March 08, 2023

On March 7, 2023, Genomic Vision, a leading biotech company that develops products and services for the analysis and control of genome changes, launched FiberSmart®, an AI-based technology for automating the quantification and detection of fluorescent signals on combed DNA molecules. Initially available for the analysis of Replication Combing Assays (RCA), Genomic Vision's proprietary method for directly visualizing DNA replication kinetics at the single molecule level. FiberSmart uses advanced AI methods to detect, visualize, and analyze DNA replication kinetics up to 3x more accurately and up to 10x faster than Genomic Vision's existing software solutions. The technology offers a simple and user-friendly interface, facilitating quick analysis of DNA replication signals to deduce essential parameters describing replication kinetics. It is compatible with Genomic Vision's FiberVision® and FiberVision-S® scanners for the RCA assay of the company's proprietary DNA combing technology with various potential applications, including in gene and cell therapy quality control. FiberSmart has been successfully tested and validated by AstraZeneca and the Fritz Lipmann Institute in Germany. Genomic Vision's CEO Aaron Bensimon said, "The launch of FiberSmart® is an important milestone for Genomic Vision as we bring the benefits of powerful AI technology to our users, who can now perform faster and more accurate genomic analysis seamlessly. Our proprietary DNA combing technique has multiple potential applications, particularly in the cell and gene therapy space, where highly accurate genomic analysis is paramount to ensure robust quality standards are met. With the launch of this software we are making it easier for users to exploit the full potential of our proprietary advanced genomic analysis technique." (Source – Business Wire) About Genomic Vision GENOMIC VISION is a leading biotechnology company specializing in the analysis of genome modifications, with a focus on ensuring their quality and safety in genome editing technologies and biomanufacturing processes. It utilizes advanced nanotechnology for DNA analysis to develop cutting-edge diagnostic and drug discovery solutions for cancer and acute diseases at the intersection of genome dynamics and human diseases. The company's approach employs Molecular Combing Technology, a powerful technique that directly visualizes single DNA molecules to detect and quantify changes in the genome landscape and their contribution to pathology.

Read More

Medical

Spotlight Therapeutics Raises $36.5 Million Series B to Advance a Pipeline of Cell-Targeted In Vivo CRISPR Gene Editing Biologics

Spotlight Therapeutics | March 22, 2022

Spotlight Therapeutics, Inc. (“Spotlight”), a biotechnology company applying new insights to develop cell-targeted in vivo CRISPR gene editing biologics, today announced a $36.5M Series B financing to fuel a drive toward the clinic. The financing round was co-led by new investors GordonMD Global Investments and EPIQ Capital Group, with participation from Magnetic Ventures, as well as existing investors GV (formerly Google Ventures) and Emerson Collective and other investors. Craig Gordon, M.D., Founder, CEO and CIO of GordonMD Global Investments, joins the Company’s Board of Directors. Spotlight's proprietary technology platform, TAGE (Targeted Active Gene Editors), is a new class of biologics; highly engineered, modular programmable CRISPR effectors designed to target and edit selected cell types in vivo. This approach circumvents the complexity of packaged viral, viral-like, and nanoparticle delivery systems, opens the door to expanded applications, and holds the promise of increasing patient access. We are excited to help Spotlight advance its pioneering work, which shows promise for cell-targeted delivery of CRISPR effectors in vivo. Spotlight’s TAGE platform could enable significant expansion of CRISPR medicines to a wide range of diseases." Dr. Gordon. This Series B funding is a crucial milestone as we advance our lead first-in-class immuno-oncology (IO) program and progress our pipeline of programs in IO, ophthalmic diseases and hemoglobinopathies,It will enable us to execute our development plan, leveraging Spotlight’s unique cell-targeted in vivo delivery approach, as we aspire to unlock the full potential of gene editing and enable effective one-and-done medicines for patients.” Mary Haak-Frendscho, Ph.D., President and CEO of Spotlight Therapeutics. About Spotlight Therapeutics Established in mid-2018, Spotlight Therapeutics is a privately held biotechnology company advancing a pipeline of cell-targeted in vivo CRISPR gene editing therapies. Spotlight's proprietary technology platform TAGE (Targeted Active Gene Editors) is a new class of biologics, CRISPR effectors engineered for direct delivery in vivo, to achieve cell-selective therapeutic genome editing. Spotlight's pipeline is advancing its modular programmable CRISPR effectors towards clinical studies in immuno-oncology, ophthalmic diseases and hemoglobinopathies. The company is headquartered in Hayward, California.

Read More

Medical

TGen Selects MemVerge to Accelerate Idiopathic Pulmonary Fibrosis Research Discovery with Big Memory Technology

MemVerge | March 21, 2022

MemVerge™, the pioneers of Big Memory software, today announced that TGen, the Translational Genomics Research Institute, an affiliate of City of Hope, has selected MemVerge Memory Machine Big Memory virtualization software to accelerate time to discovery for Idiopathic Pulmonary Fibrosis (IPF), a disease which affects 100,000 people annually in the U.S. Using MemVerge technology, TGen is able to dramatically speed analytical processing by nearly 36% for single-cell RNA sequencing. As a nonprofit medical research institute, TGen researchers process single-cell RNA sequences to characterize cell transcriptomic profiles. The process can take up to six and a half hours to analyze a matrix of 30,000 genes by 114,000 cells. With consistently growing datasets, this processing time was preventing a desired time to discovery. The data required for analysis was simply too large to retain in traditional memory, and scaling capacity with dynamic random-access memory (DRAM) was too costly. TGen has instead deployed memory virtualization technology from MemVerge which virtualizes both DRAM and PMem (persistent memory) memory technologies, to increase the memory pool available for processing without requiring more high-cost DRAM. The solution further speeds TGen's genomics sequencing analysis with Memory Machine ZeroIO in-memory snapshots which capture multi-terabyte data sets at any point for rapid reloads at each stage of processing. The ZeroIO snapshot service is 1,000 times faster than the fastest storage snapshot to SSD and enables TGen to run processing workflows in parallel. This ensures that in the event of a system crash, in-memory snapshots are available to instantly re-start long running jobs without lengthy reloading. By utilizing the snapshotting and cloning capabilities of Memory Machine, we were able to parallelize the processing workflow, As a result, we can now save nearly 36% of computational time while also taking advantage of the big memory nodes. This will save a lot of time in downstream analysis." Glen Otero, Ph.D., Vice President of Scientific Computing at TGen. MemVerge Memory Machine has quickly resulted in research value for TGen, We have removed performance barriers from their research process so that they are able to perform vital, life-saving, research faster than ever possible. Now TGen is expanding the use of Big Memory technology across other research use cases where results and discoveries can produce findings for a healthier tomorrow." Jonathan Jiang, COO of MemVerge. MemVerge Memory Machine makes 100% use of available memory capacity while providing new operational capabilities to memory-centric workloads. Memory Machine answers the need for a modern in-memory computing model to support emerging applications that require real-time analytics, true in-memory computing, and fault-tolerant memory persistence to speed massive processing workloads. About MemVerge MemVerge is pioneering Big Memory Computing and Big Memory Cloud technology for the memory-centric and multi-cloud future. MemVerge® Memory Machine™ is the industry's first software to virtualize memory hardware for fine-grained provisioning of capacity, performance, availability, and mobility. On top of the transparent memory service, Memory Machine provides another industry first, ZeroIO™ in-memory snapshots which can encapsulate terabytes of application state within seconds and enable data management at the speed of memory. The breakthrough capabilities of Big Memory Computing and Big Memory Cloud Technology are opening the door to cloud agility and flexibility for thousands of Big Memory applications.

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