Illustration of spherical nucleic acid SNA nanoparticle carrying CRISPR cargo – Image by the Mirkin Research Group University scientists in Chicago have developed a new nanostructure that triples CRISPR’s ability to enter cells, potentially unlocking even more power to treat genetic diseases. With the power to rewrite the genetic code that underlies countless diseases, CRISPR holds immense promise to revolutionize …
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CRISPR activation for SCN2A-related neurodevelopmental disorders
Karczewski, K. J. et al. The mutational constraint spectrum quantified from variation in 141,456 humans. Nature 581, 434–443 (2020). Article ADS CAS PubMed PubMed Central Google Scholar Sanders, S. J. et al. Progress in understanding and treating SCN2A-mediated disorders. Trends Neurosci. https://doi.org/10.1016/j.tins.2018.03.011 (2018). Article PubMed PubMed Central Google Scholar Maenner, M. J. et al. Prevalence of autism spectrum disorder among …
Read More »Scientists just made CRISPR three times more effective
CRISPR gene-editing machinery could transform medicine but is difficult to get into tissues and disease-relevant cells New delivery system loads CRISPR machinery inside spherical nucleic acid (SNA) nanoparticles Particles entered cells three times more effectively, tripled gene-editing efficiency, and decreased toxicity compared to current delivery methods With the power to rewrite the genetic code underlying countless diseases, CRISPR holds immense …
Read More »Cas9 senses CRISPR RNA abundance to regulate CRISPR spacer acquisition
Prokaryotes create adaptive immune memories by acquiring foreign DNA snippets, known as spacers, into the CRISPR array1. In type II CRISPR-Cas systems, the RNA-guided effector Cas9 also assists the acquisition machinery by selecting spacers from protospacer adjacent motif (PAM)-flanked DNA2,3. Here, we uncover the first biological role for Cas9 that is independent of its dual RNA partners. Following depletion of …
Read More »CRISPR can stop malaria spread by editing a single gene in mosquitos
CRISPR gene-editing therapy has shown great potential to treat and even cure diseases, but scientists are now discovering how it can be used to prevent them as well. A team of researchers found a way to edit a single gene in a mosquito that prevented it from transmitting malaria, according to a paper published in Nature. These genetically modified mosquitos …
Read More »CRISPR uncovers gene that supercharges vitamin D—and stops tumors in their tracks
Vitamin D is not only an essential nutrient, but also the precursor of the hormone calcitriol, indispensable for health: it regulates the uptake of phosphate and calcium necessary for bones by the intestines, as well as cell growth and the proper function of muscles, nerve cells, and the immune system. Now, researchers have shown for the first time in Frontiers …
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