1/3 Wonderful to finally be able to share our paper on the development and deployment of milasen, now online at It’s accompanied by thoughtful commentary from Janet Woodcock and Peter Marks of the FDA (.

RT @broadinstitute: Gene editing pioneer @davidrliu discusses baby K.J. Muldoon, the first person treated with a customized gene editing th….

RT @oligogirl: New paper out. Thanks to @irdirc N=1 taskforce members for a very positive, proactive and pleasant collaboration @N1Collabor….

Privileged to be a physician-scientist in a time when efforts like these spanning genetics, engineering, & medicine come to fruition. Hats off to @kiranmusunuru, Rebecca Ahrens-Niklas ,@UrnovFyodor & more for this milestone in interventional genetics.

RT @OTSociety: Save the date - you're invited to attend the 21st Annual Meeting of the Oligonucleotide Therapeutics Society. This event w….

RT @LaufferMarlen: David Cheerie and I will present at the first @N1Collaborative annual meeting tomorrow! We will discuss the newly establ….

RT @forebatten: Announcing Zebronkysen: Amelia & Makenzie receive personalized ASO drug. This milestone would not be possible without the i….

RT @oligogirl: Thanks to co-authors @roon_mom @LaufferMarlen @collinrwj @ypeelgersma The paper describes how @timyu & @stopbatten inspired….

RT @OTSociety: Join us for a webinar on July 25th at 8am PDT titled "Nucleic Acid Therapeutics: Successes, Milestones, and Upcoming Innovat….

RT @Sudhir_Arnay: Drugs are being approved based on antisense technology pioneered by Dr. Paul Zamecnik. My research work started in his la….

A remarkable milestone yet buried the lede: He wants to be a geneticist.

RT @davidrliu: This Thursday April 25 from 4:00-6:30 pm at the Broad Institute Julia Vitarello (Mila's mom @stopbatten), Casey McPherson (R….

RT @LaufferMarlen: Very happy that our recommendations for the identification of suitable variants for individualized ASO treatments are no….

RT @cureffi: Published today, our reasons for optimism that success rates in drug discovery can be improved by picking the right targets us….

Montage: Totality -4 min, Totality -10 sec, Totality -5 sec, 1st min of totality, 2nd min of totality, 3rd min of totality, Totality +3 sec, Totality +5 sec, Totality +2 min.

A rare treat. #Eclipse2024

RT @stopbatten: Thank you @natashaloder and The @Telegraph for sharing Mila's story and the critical role the UK is playing in proving a mo….

RT @statnews: Our first session ‘After Mila’ with @stopbatten @UrnovFyodor @Jasonmmast #STATBreakthrough

RT @OTSociety: We have many excellent resources on the website from the OTS President's perspective & educational videos, to in-depth artic….

RT @stopbatten: Today is a very exciting day. The UK government announced in the Autumn Statement by Chancellor Jeremy Hunt, their support….

RT @davidrliu: The first in vivo base editing clinical trial outcomes are now reported, following last year’s positive ex vivo base editing….