@timyu

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

theyulab.org
Joined July 2007
  • Tweets429
  • Following320
  • Followers1.9K
17Media
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…).

Patient-Customized Oligonucleotide Therapy for a Rare Genetic Disease | NEJM

Genome sequencing is often pivotal in the diagnosis of rare diseases, but many of these conditions lack specific treatments. We describe how molecular diagnosis of a rare, fatal neurodegenerative c...

nejm.org
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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
photo of David Liu

ALT photo of David Liu

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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…

Patient-Specific In Vivo Gene Editing to Treat a Rare Genetic Disease | NEJM

Base editors can correct disease-causing genetic variants. After a neonate had received a diagnosis of severe carbamoyl-phosphate synthetase 1 deficiency, a disease with an estimated 50% mortality ...

nejm.org
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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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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/…

Consensus guidelines for eligibility assessment of pathogenic variants to antisense oligonucleotide...

Of the around 7,000 known rare diseases worldwide, disease-modifying treatments are available for fewer than 5%, leaving millions of individuals without specialized therapeutic strategies. In recent...

medrxiv.org
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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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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)
AnnemiekeAartsma-Rus@oligogirl
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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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…

First Patient Begins Newly Approved Sickle Cell Gene Therapy (Published 2024)

A 12-year-old boy in the Washington, D.C., area faces months of procedures to remedy his disease. “I want to be cured,” he said.

nytimes.com
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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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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…

Practical Recommendations for the Selection of Patients for Individualized Splice‐Switching...

Although around 6% of the world’s population is affected by rare diseases, only a small number of disease-modifying therapies are available. In recent years, antisense oligonucleotides (ASOs) have...

onlinelibrary.wiley.com
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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: nitter.cf/cureffi/status/1676896…
Eric Vallabh Minikel
@cureffi
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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A rare treat. #Eclipse2024
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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
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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…

Mila’s legacy: The little girl who could change how medicines are made in Britain

Mila Vitarello was just 10 when she died from Batten’s disease. Now, the fight to find a cure for rare genetic disorders arrives in Britain

telegraph.co.uk
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Tim Yu retweeted
STAT
@statnews
21 Mar 2024
Our first session ‘After Mila’ with @stopbatten @UrnovFyodor @Jasonmmast #STATBreakthrough
Three people sitting on stage talking with the STAT Breakthrough banner in the background.

ALT Three people sitting on stage talking with the STAT Breakthrough banner in the background.

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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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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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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.
Kiran Musunuru@kiranmusunuru
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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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.
Mila's Miracle@stopbatten
26 Oct 2023
Replying to @stopbatten
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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