Neuroscience, Genetics, Genomic Medicine. Tweets my own unless one of our girls has broken into my account again in which case sorry for all the Pokémon pics
Joined July 2007
- Tweets 434
- Following 325
- Followers 1,956
- Likes 1,722
17 Photos and videos
1/3 Wonderful to finally be able to share our paper on the development and deployment of milasen, now online at nejm.org/doi/full/10.1056/NE…. It’s accompanied by thoughtful commentary from Janet Woodcock and Peter Marks of the FDA (nejm.org/doi/full/10.1056/NE…).
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Tim Yu retweeted
24 Jun 2025
Gene editing pioneer @davidrliu 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
ALT photo of David Liu
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Tim Yu retweeted
2 Jun 2025
New paper out. Thanks to @irdirc N=1 taskforce members for a very positive, proactive and pleasant collaboration @N1Collaborative @n_lorem @timyu and others!
bsky.app/profile/oligogirl.b…
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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. nejm.org/doi/full/10.1056/NE…
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Tim Yu retweeted
19 Dec 2024
Save the date - you're invited to attend the 21st Annual Meeting of the Oligonucleotide Therapeutics Society.
This event will be held on October 19-22, 2025, at the Budapest Congress Center in Budapest, Hungary.
oligotherapeutics.org/
#ots25 #oligomeeting #OTS #careers
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Tim Yu retweeted
4 Oct 2024
David Cheerie and I will present at the first @N1Collaborative annual meeting tomorrow! We will discuss the newly established N1C VARIANT (Variant Assessments towards Eligibility for Antisense Oligonucleotide Treatment) Guidelines. medrxiv.org/content/10.1101/…
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Tim Yu retweeted
25 Sep 2024
Announcing Zebronkysen: Amelia & Makenzie receive personalized ASO drug. This milestone would not be possible without the incredible support of the ForeBatten community and the passionate development team.
More information on this exciting news is on forebatten.org/
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Tim Yu retweeted
24 Sep 2024
Thanks to co-authors @roon_mom @LaufferMarlen @collinrwj @ypeelgersma The paper describes how @timyu & @stopbatten inspired us to start DCRT and lessons we have learned in our first 4 years (spoiler: most important lesson: it is important to collaborate & share @N1Collaborative)
24 Sep 2024
Our paper on the DCRT is out now in @TARareDisease
Joining forces to develop individualized antisense oligonucleotides for patients with brain or eye diseases: the example of the Dutch Center for RNA Therapeutics -
(sagepub.com)
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Tim Yu retweeted
15 Jul 2024
Join us for a webinar on July 25th at 8am PDT titled "Nucleic Acid Therapeutics: Successes, Milestones, and Upcoming Innovation."
Register now: oligotherapeutics.org/nuclei…
#nucleicacidtherapeutics #biotechnology #pharmaceuticalresearch #otswebinar #ots #nucleicacid
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A remarkable milestone yet buried the lede: He wants to be a geneticist. nytimes.com/2024/05/06/healt…
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Tim Yu retweeted
23 Apr 2024
This Thursday April 25 from 4:00-6:30 pm at the Broad Institute Julia Vitarello (Mila's mom @stopbatten), Casey McPherson (Rose's dad @ToCureARose), @The_Termeer_Fdn, @Cookies4Cures, @broadinstitute, and others will host "Rock n' Rare", a special event of inspirational stories and music celebrating family and friends of rare disease patients, scientists, doctors, and the interested public. Stories and appearances by Julia, Casey, @timyu, @SammyBasso, Dana Perella, Cole Quinn, Weston McPherson, and myself. Music by Casey McPherson and AlphaRev.
Registration: betterunite.com/rocknrare
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Tim Yu retweeted
19 Apr 2024
Very happy that our recommendations for the identification of suitable variants for individualized ASO treatments are now published!
Big thanks go to our PhD candidate Bianca Zardetto, @oligogirl, @roon_mom, and our colleagues from the @N1Collaborative hindawi.com/journals/humu/20…
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Tim Yu retweeted
17 Apr 2024
Published today, our reasons for optimism that success rates in drug discovery can be improved by picking the right targets using human genetics: nature.com/articles/s41586-0…
I break the key findings down panel by panel in this thread: x.com/cureffi/status/1676896…
6 Jul 2023
Is having even more gene-disease associations still useful for picking drug targets?
For which types of programs does it matter?
Has pharma shifted focus towards genetically validated targets?
New paper by me, @mnelsonxy, @DongCoco90417, & @jivecast doi.org/10.1101/2023.06.23.2…👇
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Tim Yu retweeted
3 Apr 2024
Thank you @natashaloder and The @Telegraph for sharing Mila's story and the critical role the UK is playing in proving a model for individualized medicines at scale.
telegraph.co.uk/global-healt…
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ALT Three people sitting on stage talking with the STAT Breakthrough banner in the background.
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Tim Yu retweeted
22 Nov 2023
We have many excellent resources on the website from the OTS President's perspective & educational videos, to in-depth articles.
View our resources on the menu under "Science News."
oligotherapeutics.org/
#ots #oligonucleotide #biotechnology #science #sciencenews
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Tim Yu retweeted
22 Nov 2023
Today is a very exciting day. The UK government announced in the Autumn Statement by Chancellor Jeremy Hunt, their support for the individualized medicines pilot MMF initiated over a year ago with support from Daniel O'Connor @parkermoss @Rich_Genomics @nicolablackwood @timyu
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Tim Yu retweeted
12 Nov 2023
The first in vivo base editing clinical trial outcomes are now reported, following last year’s positive ex vivo base editing clinical outcomes!
Congratulations to @kiranmusunuru , @skathire, @ambellinger, @NicoleGaudelli, & other scientists at @VerveTx and @BeamTx on these positive data.
12 Nov 2023
The culmination of 10 years of work to use #CRISPR gene editing to fight heart disease!
@ambellinger @VerveTx $VERV presenting the interim data from the ongoing heart-1 Phase 1b clinical trial of VERVE-101 base editing for HeFH patients, at #AHA23 now.
Let’s start!
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Tim Yu retweeted
27 Oct 2023
I’m in awe of Sonia Vallabh and Julia Vitarello, two warriors whose personal journeys and relentless commitment to connecting cutting-edge science with patients are helping to make treatments for rare genetic disease a reality.
26 Oct 2023
The bonus was sharing the stage with Sonia Vallabh who received the other OTS award, and spending the rest of the evening as fast new friends on a similar mission!
@ArtKrieg @winstonxyan @davidrliu @oligogirl @Leonard_5965 David Corey
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