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When genome editing goes off-target

Editing DNA in eukaryotic cells with CRISPR-based systems has revolutionized the genome engineering field. Cas (CRISPR-associated) endonucleases are directed to a particular location in the genome by a short guide RNA, providing an easily programmable strategy to target any section of DNA. As of now, two CRISPR-based approaches can introduce targeted, permanent edits. DNA cleavage with the Cas endonuclease facilitates small insertions or deletions of nucleotides that can disable the targeted gene (1). A second modified “base editor” system can generate precise single-base mutations in the targeted DNA (2). For both approaches, it is imperative that DNA modifications are made in the intended region (“on-target”) and not elsewhere in the genome (“off-target”). On pages 286, 289, and 292 of this issue, Wienert et al. (3), Zuo et al. (4), and Jin et al. (5), respectively, describe methods that identify off-target activities, which will be invaluable in therapeutic contexts as well as for stringent evaluation of future iterations of gene-editing tools.

Spotlight

Kameda Arabia

Kameda Arabia (KA) is a Joint Venture between Dr. Soliman Fakeeh Holding company, Jeddah and Kameda Infologics Pvt Ltd based in Japan, India and local support offices in USA, UK, UAE and Malaysia. Dr. Soliman Fakeeh is a renowned name in the Saudi Arabia Healthcare Market from past three decade. Having its Business in Healthcare, Education, Medical Item Supply's and Wellness. Kameda Infologics Pvt Ltd (KI) is a provider of integrated IT solutions to the healthcare industry and has strived to develop world-class software products specializing in Healthcare. Dr. Toshitada Kameda, founder of Kameda Informatics Ltd. is a pioneer in the development of paperless solutions and is currently rendering its service to more than 200 hospitals in Japan. It was Dr. Kameda’s vision which made Kameda Medical Center a 1000 bedded super specialty hospital, south of Tokyo, into a paperless and filmless facility since 1994. KI is using the best practices, systems and quality procedures for its software de

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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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Spotlight

Kameda Arabia

Kameda Arabia (KA) is a Joint Venture between Dr. Soliman Fakeeh Holding company, Jeddah and Kameda Infologics Pvt Ltd based in Japan, India and local support offices in USA, UK, UAE and Malaysia. Dr. Soliman Fakeeh is a renowned name in the Saudi Arabia Healthcare Market from past three decade. Having its Business in Healthcare, Education, Medical Item Supply's and Wellness. Kameda Infologics Pvt Ltd (KI) is a provider of integrated IT solutions to the healthcare industry and has strived to develop world-class software products specializing in Healthcare. Dr. Toshitada Kameda, founder of Kameda Informatics Ltd. is a pioneer in the development of paperless solutions and is currently rendering its service to more than 200 hospitals in Japan. It was Dr. Kameda’s vision which made Kameda Medical Center a 1000 bedded super specialty hospital, south of Tokyo, into a paperless and filmless facility since 1994. KI is using the best practices, systems and quality procedures for its software de

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Verantos Named "BioInformatics Company of the Year" by BioTech Breakthrough

BioTech Breakthrough, Verantos | October 28, 2021

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Industrial Impact

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Omega Therapeutics, Inc | October 14, 2021

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Verantos Named "BioInformatics Company of the Year" by BioTech Breakthrough

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Outcomes4Me Partners with Invitae to Offer Genetic Testing to Breast Cancer Patients

Outcomes4Me | October 12, 2021

Outcomes4Me Inc., developer of a leading free mobile app and platform to navigate cancer treatment and care, today announced that it has partnered with Invitae Corporation (NYSE: NVTA), a leading medical genetics company, to expand education and access to genetic testing to breast cancer patients and survivors. The collaboration leverages the strengths of Invitae, which supplies clinical grade genetic testing, and Outcomes4Me’s 360-degree, validated and evidence-based cancer support and treatment options via its free and easy-to-use app. Initially and currently available in the United States, patients can now receive genetic counseling through Invitae’s partnership with Genome Medical, get testing, and upload their results within the Outcomes4Me app.There is a misconception that genetic testing is only useful as a preventative tool prior to a cancer diagnosis. According to Outcomes4Me patient data, almost half of users (46 percent) who qualified for testing (based on NCCN Guidelines®) did not receive testing or did not know if they had received testing. However, genetic testing can provide insights that can help inform and refine precision therapy use and clinical treatment trial enrollment. In addition, genetic testing results can be used to help prevent recurrence and reduce incidence of other inherited cancers. A cancer diagnosis is often overwhelming for patients and their families. Outcomes4Me demystifies cancer by providing the most up-to-date and validated research, support, and treatment options, all grounded in science and data and curated according to the patient’s specific diagnosis. Outcomes4Me partners with the researchers, doctors, and academics that set the rigorous standards of cancer care for all treatment providers, including the National Comprehensive Care Network® (NCCN®), Vanderbilt-Ingram Cancer Center (VICC) and Massachusetts General Hospital (MGH). The collaboration with Invitae expands access to genetic testing, a vitally important tool in the patient’s cancer care arsenal. “Outcomes4Me is an indispensable platform for patients with breast cancer, giving them the personalized knowledge and access to timely new trials and targeted therapies that could lead to better health outcomes. By providing access to our comprehensive genetic testing and counseling services, Outcomes4Me is adding a valuable resource that will empower patients to advance their knowledge, understanding, and therefore, self-advocacy during treatment and survivorship.” Ed Esplin, M.D., Ph.D., FACMG, FACP, Clinical Geneticist at Invitae Unlike popular direct-to-consumer genetic testing services, which test for a few specific genetic variants for certain genes, Invitae provides state-of-the-art clinical grade next-generation sequencing-based (NGS) genetic testing that comprehensively analyses more than 80 genes, including all known mutations of the important BRCA1/BRCA2 genes. This comprehensive approach, combined with associated genetic counseling, not only provides insights for cancer patients, but also for family members who may be at risk. “Our collaboration with Invitae reinforces Outcomes4Me’s mission to give patients back control. Because of this work with Invitae, our valued community now has rare direct access to a much-needed testing service. Outcomes4Me will proudly continue to democratize the best in cancer treatment, research, and support by removing barriers and bias in information flow.” Maya R. Said, Sc. D., Founder and CEO of Outcomes4Me About Invitae Invitae Corporation (NYSE: NVTA) is a leading medical genetics company whose mission is to bring comprehensive genetic information into mainstream medicine to improve healthcare for billions of people. Invitae's goal is to aggregate the world's genetic tests into a single service with higher quality, faster turnaround time, and lower prices. About Outcomes4Me Outcomes4Me is on a mission to improve health outcomes by empowering patients with understandable, relevant and evidence-based information. Outcomes4Me has developed a platform for shared decision-making between patients and providers. The platform harnesses regulatory-grade, real-world data and patient experiences generating deeper insights and better outcomes to improve care and accelerate research. The Outcomes4Me mobile app enables cancer patients to make decisions and take control of their care based on information that is personalized to their specific condition, including finding treatment options, matching to clinical trials, and tracking and managing symptoms. Based in Boston, Massachusetts, Outcomes4Me, a woman-led company, comprises seasoned healthcare, oncology, pharmaceutical, consumer and technology veterans.

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