Personalized therapies hold tremendous promise but challenge traditional models of drug and biologic development. The @fda.gov outlines a path to market entry for products where a randomized trial is not feasible. Learn more: nej.md/4nUQdcl

After leukemia treatments failed, Alyssa Tapley, at age 13, was told she would die. Then, doctors tried an experimental gene-edited therapy. She became the first human to try the treatment made possible by U.S. federal funding. Now 16, she’s cancer-free and planning her future.

Very well done story on 60 Minutes about the harms of grant terminations and freezes at Harvard with Joan Brugge, Don Engbar, David Liu, and a very compelling young cancer patient, now cured with Liu's technology. Transcript and video here www.cbsnews.com/news/researc...

The Editor Got a Letter From ‘Dr. B.S.’ So Did a Lot of Other Editors. The rise of artificial intelligence has produced serial writers to science and medical journals, most likely using chatbots to boost the number of citations they’ve published #GiftLink www.nytimes.com/2025/11/04/s...

We then used sequence-to-activity deep learning models, to predict effects of non-coding edits on TF binding and chromatin accessibility. We first show that a ChromBPNet model can predict the same GATA site disruption mechanism exploited by the FDA-approved Casgevy medicine, specifically in T cells:

A highlight of my summer was this collab w/ @twangmdphd.bsky.social ! CRISPR base editors are driving several clinical trials, but it's hard to quantify off-target edits and their effects. Tong developed a sequencing assay to identify off-targets *in primary cells* 🧬🖥️🧪 #genomics #chromatin #CRISPR

Sensitive, direct detection of non-coding off-target base editor unwinding and editing in primary cells [new] Direct seq. quantifies off-target base editor & DNA unwinding.

Sensitive, direct detection of non-coding off-target base editor unwinding and editing in primary cells https://www.biorxiv.org/content/10.1101/2025.09.25.678665v1

1/ Happy to share our preprint from the Greenleaf Lab: beCasKAS, our method to directly detect CRISPR base editor off-targets in primary cells. We additionally show how non-coding edits can be triaged for epigenetic dysregulation using deep learning. www.biorxiv.org/content/10.1...

Delighted to share our latest work deciphering the landscape of chromatin accessibility and modeling the DNA sequence syntax rules underlying gene regulation during human fetal development! www.biorxiv.org/content/10.1... Read on for more: 🧵 1/16 #GeneReg 🧬🖥️

www.biorxiv.org/content/10.1... Our study published today on #bioRxiv describe the identification of a deaminase that converts 5mC to T, enabling direct sequencing of the human methylome and genome. This achievement was made possible through a collaborative effort across all departments at #NEB.

(1st post @BlueSky) Preprint alert🚨a long thread. Cautions in the use of @nanopore sequencing to map DNA modifications: officially reported “accuracy” ≠ reliable mapping in real applications. We performed a critical assessment of nanopore sequencing (across different versions of models) for the 1/n

who knew it would be easier to get everyone to switch social media platforms than getting them to use hg38