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A CRISPR therapy produced in record time benefits an infant with an often deadly hereditary disease, with help from UC Berkeley news.berkeley.edu/2025/05/15/inf… Children's Hospital

KJ Muldoon was born in August with a rare, life-threatening metabolic disease. Six months later, he received a CRISPR treatment made just for him to address a disease-causing misspelling in his DNA. My latest for WIRED: wired.com/story/a-baby-r…

An incredible feat...making a personalized, clinical-grade CRISPR gene therapy in 6 months that is actually efficacious! As Fyodor Urnov put it, this "is a triumph for the American peoples’ investment in biomedical research" nytimes.com/2025/05/15/hea…

Researchers led by teams at Children’s Hospital of Philadelphia and University of Pennsylvania report on the first use of the gene-editing technology CRISPR in a customized therapy designed to treat a patient with a rare disease ti.me/3GXKCCz

The first person appears to have been successfully treated with a bespoke gene-editing therapy. This breakthrough could allow such treatments to one day become a routine option for children with debilitating genetic diseases econ.st/431MrqA

Eric continues to be his incomparable self in lucid, accurate, accessible descriptions of major biomedical advances. (in this case I'm biased since I was on the team)

Interview: Moving Therapeutic Genome Editing into Global Clinical Trials and Medicine -- A timely interview with Kiran Musunuru and Fyodor Urnov Global Observatory for Genome Editing liebertpub.com/doi/10.1089/cr…

.Jason Mast provides a clear-eyed summary of how we are getting from N=1 (Baby KJ) to N=many. STAT statnews.com/2025/05/26/wha…

Ten years ago a piece in Nature entitled "Do Not Edit the Human Germline" authored by yours truly and colleagues argued against this dangerous and unnecessary application of editing. I thank the august lineup of authors of this timely piece for taking a strong stand on this.

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.

Baby KJ, the world's first patient to receive personalized CRISPR gene editing therapy for a rare metabolic disease, is now home and thriving! This groundbreaking treatment offers hope for other untreatable genetic conditions. #CRISPR #GeneEditing yahoo.com/news/worlds-fi…

After speaking with Baby KJ's physician and parents Secretary Kennedy said today: "CRISPR gives the gift of life." My remarks to HHS, FDA, CMS, NIH, and CBER leadership are below: we can and we must provide that gift of life to all children who need it. youtube.com/watch?v=0qDhRt…

My entire generation grew up on that legendary rotascoped video. Still a sui generis. My CRISPR cures mind now refilms it where he gets rescued by a doctor who gives him a bespoke medicine. nytimes.com/2025/06/04/art…

.Secretary Kennedy said yesterday at the FDA: "CRISPR gives the gift of life." He is 100% correct. I appreciate HHS, FDA, CMS, NIH, and CBER leadership devoting their morning to communicate their clear support for CRISPR giving such a gift to all of our nation's children who need it.

This FDA is an FDA interested in listening and learning and evolving, not just our thinking in regulation, but also our processes at the FDA. Here's more from yesterday's FDA Cell and Gene Therapy Roundtable. x.com/US_FDA/status/…

Anyone interested in, or developing, gene editing medicines is well advised to watch this video. In it, HHS, FDA, CMS, and NIH leadership affirm their support for scaling up such medicine development in the US, and the key role NIH-supported biomedical research plays in it.

"At the end of every hard-earned day People find a reason to believe" (Springsteen) Watch this video. It's data-based reason to believe. Don't forget Kleenex. I watched this video 4 times and cried every single time.

This. Is. Not. A. Dire. Wolf. Not. Even. Close. The CSO of Colossal has publicly stated as much (she is a scientist). No amount of salesmanship by the business people behind Colossal can change laws of biology. Enjoy video of a cute wolf puppy and savor being science-savvy.

Watch. Weep tears of joy. Get inspired.