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latest CRISPR news

𝐀𝐧𝐨𝐭𝐡𝐞𝐫 𝐅𝐫𝐢𝐝𝐚𝐲, 𝐚𝐧𝐨𝐭𝐡𝐞𝐫 𝐩𝐚𝐜𝐤𝐞𝐝 𝐧𝐞𝐰𝐬𝐥𝐞𝐭𝐭𝐞𝐫 𝐟𝐫𝐨𝐦 𝐂𝐑𝐈𝐒𝐏𝐑 𝐌𝐞𝐝𝐢𝐜𝐢𝐧𝐞 𝐍𝐞𝐰𝐬! ✂ Exciting results from Stephanie Cherqui at UC San Diego and colleagues at Sanford Consortium for Regenerative Medicine, who demonstrate that excision of certain repeats in the FRX gene can rescue disease phenotypes in Friedreich’s ataxia iPSC-derived neurons. ✅ The European Commission approves CASGEVY™ for the treatment of sickle cell disease and beta thalassemia in individuals 12 years and older. 🤝 Intellia Therapeutics, Inc. and ReCode Therapeutics join forces to develop new gene-editing therapies for cystic fibrosis. Get all the news below 👇 https://lnkd.in/ezvnWxHA #crisprmedicinenews #crisprmedicine #geneediting #fda #crispr #raredisease #clinicalnews #crisprnews #crisprtrial Sangamo Therapeutics, Inc. CRISPR Therapeutics Fate Therapeutics Inc Precision BioSciences, Inc. Prime Medicine, Inc.

This study evaluated the effect of candidate genes and a novel CYP2C haplotype on voriconazole trough concentrations in patients receiving CYP2C19-guided dosing. CYP2C19 phenotype and letermovir use were significantly associated with subtherapeutic voriconazole concentrations and may be used to improve voriconazole precision dosing, while further research is needed to clarify the role of ABCG2 in voriconazole dosing. Read more via The Pharmacogenomics Journal: https://lnkd.in/ghPUqv6U | #Pharmacogenomics #PrecisionMedicine #PrecisionDosing #Voriconazole

Find your target. Our functional genomic tools enable comprehensive screening of potential targets, while our cell culture tools help you validate using the most predictive model for your project. Explore more on our mAb target discovery pages. #mAbs

𝗥𝗡𝗔 𝗲𝗱𝗶𝘁𝗶𝗻𝗴 𝗶𝘀 𝗴𝗮𝗶𝗻𝗶𝗻𝗴 𝘀𝗽𝗲𝗲𝗱, 𝗲𝘀𝗽𝗲𝗰𝗶𝗮𝗹𝗹𝘆 𝗳𝗼𝗿 #𝗿𝗮𝗿𝗲 𝗱𝗶𝘀𝗲𝗮𝘀𝗲 RNA editing is a new gene-editing technology that has entered clinical trials recently. It is believed to offer safer and more flexible gene-editing therapies. There are two RNA editing treatments currently being tested   💊 The first treatment by AIRNA focuses on RNA editing to make more precise medicines, using ADAR. The treatment replaces a single base to correct a mutation that leads to alpha-1 antitrypsin deficiency (#AATD). #AATD is a condition that can harm the lungs and liver.  https://lnkd.in/dN67F7AV    💊 The second treatment by Ascidian Therapeutics Therapeutics (Rewriting RNA) changes thousands of genetic letters in an RNA molecule simultaneously. Recently, FDA cleared the first Clinical Trial of #RNA Exon Editor Developed to Treat #Stargardt Disease, and other retinal diseases caused by mutations in the ATP binding cassette transporter gene, ABCA4. This treatment aims to treat the group of mutations that cause Stargardt disease. https://lnkd.in/d77AYwnk Kris Elverum , Michael Ehlers https://lnkd.in/d9z7sK4s

This image entitled 'The Glomerulus in Chronic Antibody Mediated Rejection' shows how CosMx SMI was used to analyze human allograft kidney core biopsies with chronic antibody-mediated rejection (CAMR). This glomerulus image was obtained from a case of CAMR with transplant glomerulopathy and shows the glomerular endothelial cells (GECs) and podocytes identified using specific gene sets. The GECs are adjacent to podocytes. Credit: Ivy Rosales, Massachusetts General Hospital Curious to know the latest on CosMx SMI? Check out our AGBT webinar: https://bit.ly/48JN6gO #SaturdaySlideShow #spatialsinglecell #kidneytransplant This post may include information regarding CosMx™ SMI products for RNA detection, which products are not available in the member states of the European Unified Patent Court, as further described here - https://bit.ly/498JPIB.

Intracellular protein detection from single cells? With whole transcriptome sequencing and cell surface protein detection? And with full length TCR/BCR sequencing? And sample multiplexing? YES, with Rhapsody platform. Maximise single cell data collection from your precious samples. #singlecellanalysis #multiomics #Immunology #CITE-seq

Good review of different pathways to cell death, and consequences of each type of death.

Very nice review that covers cell death pathways and touches upon dead cell elimination. Understanding the consequences of a cell death pathway in terms of the type of debris generated and the mechanisms for clearing that debris (ex. inflammatory vs non-inflammatory) is as important to drug discovery as understanding the cell death pathway itself. Ideally all three elements, cell death pathway, debris and clearance mechanisms, should be incorporated into a biomarker strategy that is indicative of drug action independent of efficacy in early development to know if, when and how to progress treatment.

Find your target. Our functional genomic tools enable comprehensive screening of potential targets, while our cell culture tools help you validate using the most predictive model for your project. Explore more on our mAb target discovery pages. #mAbs