'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 nejm.org/doi/full/10.10… nytimes.com/2022/06/05/hea… #ASCO22

It's been a huge privilege to do my DPhil alongside fantastic labmates, collaborators, and mentors at MRC WIMM and Department of Paediatrics. Pushing the last mile for the paper and can't wait to bring this tech from bench to bedside. Sauka-Spengler Lab Carlo Rinaldi RDM Oxford

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. science.org/doi/10.1126/sc…

Genome editing for potential definitive treatment or curies for many genetic conditions is moving forward at high velocity. A new Cell review is a masterful state-of-the-science for delivery cell.com/cell/fulltext/… David R. Liu Samagya Banskota liugroup Broad Institute

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) nature.com/articles/s4158…

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! docs.google.com/document/d/17n…

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. biorxiv.org/content/10.110…

How did idea for Verve Therapeutics come about? 👇 (L) April 2016, our response to American Heart Association 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 😊

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

Delighted to announce that our portfolio company MiroBio is to be acquired by Gilead Sciences for approximately $405 million. Read our full press release to learn more ⬇ bit.ly/3zzzJ2i

Today we share in Nature Protocols detailed strategy and experimental procedures for designing and executing prime editing (and twinPE) experiments in mammalian cells. Congrats Jordan, Alexander Sousa, Peyton, and Peter Chen for this thorough analysis. drive.google.com/file/d/12oIqq5…

CRISPR can cut DNA and RNA, but did you ever wonder other activities might exist? Today in bioRxiv, we show that the Type III-E Cas7-11 system programmably activates a nearby protease as part of microbial defense. + Jonathan Gootenberg hnisimasu 🧵👇🏼biorxiv.org/content/10.110… 1/9

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 PhD Voice - Independently Run Academic Chatter™

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

Fantastic study from ⁦MarineLab⁩ 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. nature.com/articles/s4158…

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

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.

Today we report in Science Magazine 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. science.org/doi/10.1126/sc… PDF: drive.google.com/file/d/1H1k8AA… 1/17