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CHOP & Penn Medicine have delivered the first-ever personalized gene editing therapy for a patient with CPS1 deficiency, marking a major milestone in the application of CRISPR-based treatments. Learn more about KJ & the future of personalized medicine: ms.spr.ly/6015SZLAx.

Breaking News: A baby with a rare disorder made medical history by receiving the first custom gene-editing treatment. The technique used has the potential to help people with thousands of other uncommon genetic diseases. nyti.ms/4j3xGs8

Today "a milestone in the evolution of personalized therapies for rare & ultra-rare inborn errors of metabolism" —the 1st human to undergo custom genome editing —outgrowth of decades of NIH funded research nejm.org/doi/full/10.10… nejm.org/doi/full/10.10… NEJM

In a medical milestone, a customized base editor was developed, characterized in human and mouse cells, tested in mice, studied for safety in non-human primates, cleared by U.S. FDA for clinical trial use, manufactured as a complex with an LNP, and dosed into a baby with a severe,

A baby named KJ, who was born with a rare genetic disorder – CPS1 deficiency – is healed thanks to doctors at the Children's Hospital who administered the world’s first personalized gene-editing treatment. The research team used base editing, a gene-editing method invented by HHMI

Never could I have imagined that ABE would someday enable such a transformative outcome for a child. Thank you Kiran Musunuru, Rebecca Ahrens-Nicklas, and KJ’s family for having the courage to demonstrate to the world what is possible. Incredible. 💕 nytimes.com/2025/05/15/hea… via

A triumph of perseverance from twitterless Tom Weber, Christine Biben and the team, our in vivo barcoding "LoxCode mouse" used to resolve epiblast fate to fetal organs is finally published in Cell and available to import through The Jackson Laboratory sciencedirect.com/science/articl…

CloneSelect published in Nature Biotechnology. Please check out this GIFtorial. This retrospective clone isolation method using CRISPR base editors is a powerful tool in broad biology. A history of Soh Ishiguro in yachielab. nature.com/articles/s4158… UBC School of Biomedical Engineering 東京大学先端科学技術研究センター 大阪大学PRIMe

Honored to help lead the Global Observatory Summit — our biggest yet. Scientists, ethicists & artists gathered to ask not just what #CRISPR can do, but what it should do. We closed with a call for a global charter on tech & human dignity. 📰STAT statnews.com/2025/05/27/glo…

Join us this Thursday for FDA's Cell and Gene Therapy Roundtable. You'll be able to view the livestream from 9am - noon right here on X U.S. FDA.🎥fda.gov/vaccines-blood…

In case you needed a smile this Tuesday: 10-month-old baby KJ, who received a bespoke gene-editing therapy after a six month scientific sprint - "graduated" from the hospital today and went HOME! YAY SCIENCE! Here's his story: wapo.st/451ZQjT

No words - only tears of joy. And bow of gratitude to Rebecca Ahrens-nicklas (CHOP) and Kiran Musunuru (Penn) and Sadik Kassim (Danaher) - our amazing partners in designing, derisking, and administering a CRISPR on demand to Baby KJ.

LIVE NOW: FDA's Cell and Gene Therapy Roundtable Captions available here: bit.ly/4dQaNaz x.com/i/broadcasts/1…

After this week’s cell and gene therapy roundtable at U.S. FDA, Secretary Kennedy joined DrOzCMS, Dr. Martin Makary, and Jay Bhattacharya, MD, PhD to deliver a message directly to the American people about investing in cutting-edge cures. Cell and gene therapy holds promise—but it also demands

Phage metagenomes hold powerful secrets! This article breaks down our recent work using Meta-SIFT to uncover functional motifs hidden in viral genomes—and how we used them to engineer phages that kill bacteria responsible for food-borne outbreaks. Science Advances Phenomenal

Gene editing pioneer David R. Liu discusses baby K.J. Muldoon, the first person treated with a customized gene editing therapy, what made K.J.'s treatment possible, and how on-demand treatments for rare genetic diseases could one day become routine. broad.io/DLQA

Thank you Somatic Cell Genome Editing (SCGE) Outreach for the opportunity to speak to the consortium about alternative commercialization models on today's Meet the Expert webinar! So great to talk about our work at Orphan Therapeutics Accelerator with so many familiar faces 😊