Today we report in @ScienceMagazine a one-time base editing treatment for Spinal Muscular Atrophy (SMA), the leading genetic cause of infant mortality worldwide, affecting ~1 in 10,000 births. https://t.co/oLp8VN0Iiw PDF: https://t.co/JoKm9BxW89 1/17

A clever strategy to build a synthetic 'organelle' in prokaryotes to run a custom biological pathways. Also it started as an iGEM project! They really show you how far iGEM projects can go.

Our report of first-in-human GPRC5D-targeted CAR T cells for MM is out in NEJM! The trial was led by Sham Mailankody and could not be done without the amazing clinical, correlative science, stats, & cell manufacturing teams. Happy to share our bench>bedside journey 1/10

Fantastic study from ⁦@lab_marine⁩ on the cellular hierarchy that governs melanoma growth and metastasis demonstrated by lineage tracing. Congratulations to all the authors and very honored to be part of this beautiful study.

Are you 'Excited to share my new paper!' Harsh truth: Most people don't care 5 tips on how to tweet about your new paper to get real engagement @OpenAcademics @PhD_Genie @PhDVoice @AcademicChatter

Enjoyed this🧵? Please RT the first tweet. And follow me @LNivisonSmith if you are interested in - macular degeneration research - #WomeninSTEM - #scicomm - musings from a mother trying to survive #academia

CRISPR can cut DNA and RNA, but did you ever wonder other activities might exist? Today in @biorxivpreprint, we show that the Type III-E Cas7-11 system programmably activates a nearby protease as part of microbial defense. + @jgooten @hnisimasu 🧵👇🏼 https://t.co/QIg548Jjy8 1/9

Today we share in @NatureProtocols detailed strategy and experimental procedures for designing and executing prime editing (and twinPE) experiments in mammalian cells. Congrats Jordan, @AlexanderASousa, Peyton, and @PeterJ_Chen for this thorough analysis. https://t.co/ZlsYqaVud1

Delighted to announce that our portfolio company @Miro_Bio is to be acquired by @GileadSciences for approximately $405 million. Read our full press release to learn more ⬇ https://t.co/9L3abxLxrS

RNA therapeutics landscape (private and public). From Cowen's recent RNA review.

How did idea for @VerveTx come about? 👇 (L) April 2016, our response to @American_Heart One Brave Idea grant competition looking for coronary heart disease cure (R) Our proposed timeline from then, dose first patient in ~5y And (despite not getting award), right on time 😊

Preprint alert 🚨: #AAV vectors are one of the leading platforms for gene delivery for the treatment of human genetic diseases. Despite some clinical successes, AAV-based gene therapy still faces several challenges.

The cost of living crisis in the UK has severely impacted post-graduate researchers. This is an open letter to the UKRI petitioning for the support of an increase to their stipend. Please support and share! https://t.co/tElVolgeLz

Such an interesting time for biotech and medicine

15 years ago, probably no one would have thought that we will have a therapeutic gene editing by 2022. But here we are, with Verve and Beam testing a drug that could edit a part of the human genome to give people a 'superpower' (i.e. lower risk of cardiovascular events).

Genome editing for potential definitive treatment or curies for many genetic conditions is moving forward at high velocity. A new @CellCellPress review is a masterful state-of-the-science for delivery https://t.co/SJtJ512NE1 @davidrliu @samagyabanskota @liugroup @broadinstitute

The peer-reviewed version of DNA Ticker Tape (now “DNA Typewriter”) is out today! During revision, we have expanded our lineage recording demonstration by integrating DNA Tape recording with single-cell RNA-seq (1/8) https://t.co/wVKkT5CL4f

Myotonic dystrophy type 1 (DM1) is the most common form of muscular dystrophy but the molecular basis for neurocognitive defects in DM1 patients is unknown. Excited to share our cover work where we generated 3D cortical organoids from DM1 patients. https://t.co/f5AxFwi4fG

It's been a huge privilege to do my DPhil alongside fantastic labmates, collaborators, and mentors at @MRC_WIMM and @OxPaediatrics. Pushing the last mile for the paper and can't wait to bring this tech from bench to bedside. @TSS_Lab @rinaldi_ca @RDMOxford

'A complete remission in every single patient is “unheard-of”' Combining genomics diagnosis of mismatch repair deficient + PD-1 immunotherapy led to complete remission of 18 consecutive patients w/ advanced colorectal cancer https://t.co/54yFGoTDoZ https://t.co/CZsoFHM0Gl #ASCO22