People With Severe Diabetes Are Cured in Small Trial of New Drug nytimes.com/2025/06/20/hea… via @NYTimes

Lipid nanoparticle screening in nonhuman primates with minimal loss of life bit.ly/3I0TO9A

🚨🚨🚨Big news for development of in vivo CAR T cells: AbbVie (ABBV) to Acquire Capstan Therapeutics for $2.1 Billion AbbVie Capstan Therapeutics #TCell #tcellrx #immunotherapy streetinsider.com/Corporate+News…

The first cancer vaccines matched to a person’s unique tumor neoantigens are forging ahead, with expectations running high as the field awaits results from the first pivotal trial nature.com/articles/s4158… rdcu.be/euaoK

This week we publish in Nature Biotechnology "High-precision cytosine base editors by evolving nucleic-acid-recognition hotspots in deaminase". In this work, we show that both nucleotide and context specificity of deaminases can be reprogrammed by hotspot-focused directed evolution 1/n

Most studies on clinical utility of exome sequencing in intensive care focussed on pediatric patients. In a new study in AJHG, the authors assessed diagnostic value of exome sequencing in critically ill adult patients and found a surprising 24% diagnostic yield, many with

🧵1/Today, we published a key milestone towards AI based cardiac screening in Nature. doi.org/10.1038/s41586… EchoNext outperformed cardiologists and found thousands of high-risk patients missed in routine care. We also made a version available to the world.

A pioneering reproductive technology reduced the risk of children inheriting disease-causing mutations in mitochondria, researchers reported. trib.al/6nwY1Vx

The past 97 years of 285 Penn - originally an Academy at Fourth and Arch Streets, then moving to 9th and Market in 1829, then to West Philadelphia in 1872

Today in Cell we report the use of prime editing to correct several mutations that cause alternating hemiplegia of childhood (AHC), a rare and devastating neurodevelopmental disorder, in patient-derived cells and in two mouse models. drive.google.com/file/d/1ibC50t… 1/10

🚨Just Announced: The 2025 ASHG Professional Award Winners! Meet the innovators shaping the future of human genetics. Johns Hopkins Medicine, MassGeneral News, Penn Medicine, CWRU School of Medicine, CU Anschutz Medical Campus, The University of Chicago 💥Full list & details: ashg.org/membership/awa… #ASHG #HumanGenetics

We’re excited to share new data published in nature detailing the impressive activity, specificity, and low immunogenicity of our AI-designed CRISPR-Cas proteins, including OpenCRISPR-1. The future of gene editing is here and we’re scaling our capabilities to tackle the

Data from the second human treated with a prime edited medicine: a 56-year-old male with chronic granulotamous disease (CGD) had his bone marrow prime edited to insert the missing GT in NCF1, restoring NADPH oxidase function in neutrophils (DHR positivity) several fold beyond the

Congrats to Rebecca Ahrens-Nicklas for being named Rad Girl of the Year at the 2025 Rad Awards! Dr. Ahrens-Nicklas was nominated for her groundbreaking work in personalized gene therapy with baby KJ. Learn more about the 2025 Rad Award winners at thephiladelphiacitizen.org/2025-rad-award…

Once a Death Sentence, This Heart Condition Is Finally Treatable nytimes.com/2025/08/04/wel…

A wonderful article by Dhruv Khullar on patients with progeria, the genetics behind this rare disease, and our ongoing efforts with PRF and NIH to advance promising base editing outcomes in humanized progeria mice into a clinical trial. newyorker.com/magazine/2025/…

This is a cool paper, but not sure if the findings are impressive enough to be too excited about. The authors used a GWAS of a pain phenotype and decided to study a gene (SLC45A4) at a specific locus in detail. The genetic association itself is robust, replicates across

🚨 2025 Focused Update of the 2019 ESC/EAS Guidelines for the management of dyslipidaemias #Cardiology #ESC2025 #LipidUpdate 1️⃣ NEW cardiovascular risk estimation: Use updated #SCORE2 & #SCORE2OP algorithms for 10-year risk in adults aged 40–89, calibrated to local mortality

Are A.I. Tools Making Doctors Worse at Their Jobs? nytimes.com/2025/08/28/wel…

Most genetic reports label variants as ✅ “pathogenic” or ❌ “benign.” But what patients really want to know is: what are my chances of getting the disease? 🚨 In our new Science study, we used AI + 1.3M patient records to better answer that…