Medicine
fromNature
3 days agoPersonalized CRISPR therapies could soon reach thousands - here's how
FDA proposed a 'plausible mechanism pathway' to enhance development of personalized genetic therapies for rare disorders.
The UK Biobank has become an important resource for researchers who, since 2012, have been able to request access to anonymised data in order to examine the causes, prevalence and treatment of myriad diseases.
Using CRISPR-Cas9 and adeno-associated virus (AAV)-mediated homology-directed repair, we targeted CAR integration into the endogenous human TCR alpha locus (TRAC). TRAC-CAR T cells display dynamic CAR expression that delays exhaustion and improves tumour control in xenograft and immunocompetent models. This work has been critical for the development of allogeneic CAR T cell therapy, as it disrupts the TCR after transgene insertion—a necessary step to limit graft-versus-host disease.
Small organic molecules underpin modern life, from medicines and flavours to advanced materials. Much of this functional diversity comes from shape: modest changes in a molecule's 3D structure can completely change its properties.
It was another detail that the rest of the family apparently knew but had never told me; they thought I already knew. The biology mattered less to me than the secret. Dad had been adopted, it turned out. A classic affliction of the 1950s, in which young, unmarried couples were forced to give away their newborn babies.
The strategy could be fruitful: If unidentified DNA evidence can be connected to someone - even a distant relative - in a common genealogy database, it would give investigators more information and possibly lead to a suspect in Guthrie's kidnapping in Arizona. "It's a fantastic tool," said Ruth Ballard, a geneticist in California who specializes in DNA and has testified in hundreds of court cases. "If it's a good quality sample and they're able to get a profile, they could find a hit on that fairly quickly."
Scientists in the laboratory of Rendong Yang, PhD, associate professor of Urology, have developed a new large language model that can interpret transcriptomic data in cancer cell lines more accurately than conventional approaches, as detailed in a recent study published in Nature Communications. Long-read RNA sequencing technologies have transformed transcriptomics research by detecting complex RNA splicing and gene fusion events that have often been missed by conventional short-read RNA-sequencing methods.
Now, researchers have created an artificial-intelligence system that vastly simplifies and accelerates the process of chemical synthesis. The system, which is called MOSAIC and is described in a study published in Nature on 19 January, recommended conditions that researchers were able to use to generate 35 compounds with the potential to become products like pharmaceuticals, agrochemicals or cosmetics without needing to do any further trawling or tweaking.
Martschenko's argument is largely that genetic research and data have almost always been used thus far as a justification to further entrench extant social inequalities. But we know the solutions to many of the injustices in our world-trying to lift people out of poverty, for example-and we certainly don't need more genetic research to implement them. Trejo's point is largely that more information is generally better than less.
Biology is undergoing a transformation. After centuries of studying life as it evolves naturally, researchers are now using a combination of computation and genome engineering to intervene, generating new proteins and even whole bacteria from scratch. The use of artificial-intelligence tools to design biological components, an approach known as generative biology, is set to turbocharge this area of research. Just last year, scientists used AI-assisted design to produce artificial genes that can be expressed in mammalian cells.
GEMINI leverages a computationally designed protein assembly as an intracellular memory device to record the history of individual cells. GEMINI grows predictably within live cells, capturing cellular events as tree-ring-like fluorescent patterns for imaging-based retrospective readout. Absolute chronological information of activity histories is attainable with hour-level accuracy.