The model can diagnose problems as well as a human specialist, and doesn’t need lots of labor-intensive training data.
The National Institutes of Health will invest $130 million over four years, pending the availability of funds, to accelerate the widespread use of artificial intelligence (AI) by the biomedical and behavioral research communities. The NIH Common Fund’s Bridge to Artificial Intelligence (Bridge2AI) program is assembling team members from diverse disciplines and backgrounds to generate tools, resources, and richly detailed data that are responsive to AI approaches. At the same time, the program will ensure its tools and data do not perpetuate inequities or ethical problems that may occur during data collection and analysis. Through extensive collaboration across projects, Bridge2AI researchers will create guidance and standards for the development of ethically sourced, state-of-the-art, AI-ready data sets that have the potential to help solve some of the most pressing challenges in human health — such as uncovering how genetic, behavioral, and environmental factors influence a person’s physical condition throughout their life.
“Generating high-quality ethically sourced data sets is crucial for enabling the use of next-generation AI technologies that transform how we do research,” said Lawrence A. Tabak, D.D.S., Ph.D., Performing the Duties of the Director of NIH. “The solutions to long-standing challenges in human health are at our fingertips, and now is the time to connect researchers and AI technologies to tackle our most difficult research questions and ultimately help improve human health.”
Intellia Therapeutics said Friday the first six patients to receive its CRISPR-based treatment for a genetic swelling disorder have safely had small, corrective changes made to dysfunctional DNA inside their liver cells.
Preliminary results from the study — just the second to show that CRISPR-based gene editing can be delivered systemically and performed in vivo, or inside the body — found that the treatment, NTLA-2002, reduced levels of the disease-causing protein, kallikrein, by 65% and 92% in the low-and high-dose cohort, respectively. In the low-dose group, the one-time infusion also reduced by 91% the painful swelling “attacks” commonly experienced by patients with a rare condition called hereditary angioedema, or HAE. Participants in the high-dose group have not yet completed the 16-week observation period.
The research community is excited about the potential of DNA to function as long-term archival storage. That’s largely because it’s extremely dense, chemically stable for tens of thousands of years, and comes in a format we’re unlikely to forget how to read. While there has been some interesting progress, efforts have mostly stayed in the research community because of the high costs and extremely slow read and write speeds. These are problems that need to be solved before DNA-based storage can be practical.
So we were surprised to hear that storage giant Seagate had entered into a collaboration with a DNA-based storage company called Catalog. To find out how close the company’s technology is to being useful, we talked to Catalog’s CEO, Hyunjun Park indicated that Catalog’s approach is counterintuitive on two levels: It doesn’t store data the way you’d expect, and it isn’t focusing on archival storage at all.
CAR-T immune therapies were developed to fight cancer. The same technique just showed promise treating lupus, but it costs hundreds of thousands.
Circa 2008 face_with_colon_three
Adult bone marrow (BM) houses a tiny pool of hematopoietic stem cells (HSCs) that have the ability to maintain not only themselves but also all the rest of highly turning over blood lineages throughout the mammalian life (1, 2). Hence, the ability to sustain HSC in tissue culture would allow serial introduction of gain or loss of function mutations efficiently in hematopoietic system. However, our failure to expand HSC in culture has hampered the use of this approach. In fact, BM suspension cultures lose rapidly their HSC content despite vigorous growth of progenitors and more differentiated cells at least for 3 weeks even in optimal cytokine milieu (3, 4). Therefore, the phenomenon of stem cell exhaustion or senescence may set the limits that make it impossible even in principle to expand HSC in culture for longer periods (5–7).
Mouse HSC do expand in vivo (8, 9), at least up to 8000-fold, as shown by Iscove and Nawa (9) through serial transplantation experiments that assessed carefully the input and output contents of HSC in each transfer generation. Recently also in vitro approaches have been improved and refined culture conditions with new growth factors can now support up to 30-fold expansion of mouse HSC ex vivo (10). However, since it is not clear to what extent external culture conditions can be improved, alternative but not mutually exclusive efforts to change the intrinsic properties of HSC have been taken. Seminal experiments in this respect by Humphries, Savageau and their colleagues have shown that ectopic expression of HOXB4 transcription factor in BM cells support the survival and expansion of HSC in vivo and importantly also in vitro (11–13). By rigorously monitoring the HSC content in their cultures of HOXB4-transduced BM cells, they found that HSC could be expanded up to 41-fold in the 2-week liquid cultures (13). HOXB4 belongs to a large family of HOX transcription factors that are crucial for the basic developmental processes in addition to their role in maintenance of different stem cell compartments.
Continue reading “Manipulation of immune system via immortal bone marrow stem cells” »
Chardan hosted its 4th Annual Chardan Genetic Medicines Conference in October 2020, featuring over 80 public and private companies representing in vivo gene therapy, ex vivo gene therapy, gene editing, RNA medicines, and other subsegments of the genetic medicines space. Among our various panels with preeminent thought leaders, we spoke with newly-minted Nobel laureate, President of the Innovative Genomics Institute, and Professor of Molecular and Cell Biology and Chemistry at UC Berkeley, Jennifer Doudna.
PhD about open questions and areas of innovation in the CRISPR gene editing space.
A study on naked mole rats could help scientists prevent and better treat human illnesses.
According to new research conducted by University of Cambridge scientists, naked mole rats age healthily, very rarely get cancer, and are numb to acid.
The team hopes to utilize these insights to find better treatment methods for human illnesses and inflammatory conditions such as arthritis, according to an institutional press release.
A new autoimmune therapy harnesses a person’s own cells to find and correct other defective cells – an answer for patients who haven’t responded to other treatments and a possible cure for diseases like lupus.
Autoimmune diseases are conditions where the body’s immune system attacks other cells in the body and causes symptoms like inflammation. This happens through autoantibodies, or antibodies that attack the self, produced by a type of white blood known as B cells.
New research tests a type of immunotherapy where T cells, another type of white blood cell, are edited to root out the defective B cells.
Circa 2019 face_with_colon_three
The most widely used methods for transdermal administration of the drugs are hypodermic needles, topical creams, and transdermal patches. The effect of most of the therapeutic agents is limited due to the stratum corneum layer of the skin, which serves as a barrier for the molecules and thus only a few molecules are able to reach the site of action. A new form of delivery system called the microneedles helps to enhance the delivery of the drug through this route and overcoming the various problems associated with the conventional formulations. The primary principle involves disruption of the skin layer, thus creating micron size pathways that lead the drug directly to the epidermis or upper dermis region from where the drug can directly go into the systemic circulation without facing the barrier. This review describes the various potential and applications of the microneedles. The various types of microneedles can be fabricated like solid, dissolving, hydrogel, coated and hollow microneedles. Fabrication method selected depends on the type and material of the microneedle. This system has increased its application to many fields like oligonucleotide delivery, vaccine delivery, insulin delivery, and even in cosmetics. In recent years, many microneedle products are coming into the market. Although a lot of research needs to be done to overcome the various challenges before the microneedles can successfully launch into the market.
