In a major milestone for science, for medicine, and for patients, the world’s first CRISPR drug has been approved in the UK.
“A new treatment for sickle-cell disease and transfusion-dependent β-thalassemia has been authorised by the Medicines and Healthcare products Regulatory Agency (MHRA) for patients aged 12 and over after a rigorous assessment of its safety, quality and effectiveness.”
Congratulations to the many scientists, doctors, patients, and industry leaders who made this development possible.🍻
https://t.co/pUL956YsG3
Screening preselected presenter (FKBP12) and target (mTOR) proteins to discover molecular glues exemplified by the discovery of a synthetic mimic of rapamycin https://t.co/PXtS3Mjm2h
🔥🔥 Exciting developments on novel quality control mechanisms by the UBR5 E3 ubiquitin ligases. Potential opportunity for molecular glue discovery to take on some challenging transcription factors. Congrats @RapeLab@LabThoma et al.
🎯Targeted protein degradation as a therapeutic strategy: the focus of the 39th #BarcelonaBioMed Conference.
🗣The @FundacionBBVA & #IRBBarcelona are bringing together in #Barcelona more than 150 scientists from 🌏all over the world to discuss the latest advances in this promising field.
➡️https://t.co/AzxQquWd7a
#TPD
The career question I get asked the most is “why did I become a Physician-Scientist” - too often I give a vanilla answer to this - some colleagues have a Eureka moment- for me it was uncertainty, failure & extraordinary mentorship that set me on my journey https://t.co/RpTHgDuwCM
Today, we publish a paper in @Nature that describes how the DNA-binding protein CTCF regulates DNA loop formation by cohesin.
This is a collaboration with Jan-Michael Peters' lab at @IMPvienna, greatly led by 1st authors Iain Davidson (Vienna) & @RomanBarth2 (CDlab at Delft)
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How can you uncover novel biological mechanisms beginning with observations from human genetics? Read this:
Large-scale variant-to-function mapping in primary hematopoietic stem and progenitor cells from @TheXavierLab
https://t.co/kIyMN0e3gZ
More fireworks after 6 pm at #AACR23: #CICR townhall with 3 awesome talks about molecular glue degraders. Michael Rapé (@RapeLab), Riccardo Sabatini (@OrionisBio) & Mary Matyskiela (@neomorphinc). Chaired by Ben Ebert (@DFCI_ChemBio). Glueing everybody to their seats. @AACR#TPD
1/2. Lots of mysteries surrounding glue mechanisms, so its nice to see hydrophobic shielding of hydrogen bonds highlighted here by @Dereklowe. I'll bet there will be other surprises uncovered in the future.
https://t.co/K3NpIJ5GXL
Front line role of our leading cancer researchers at #AACR23 in tackling one of cancers holy grails - targeting the RAS oncogene @bensolomon1#JayeshDesai https://t.co/GgTUCXTK3p
🧵Grateful to share our work @JAMAOnc@DrDanielHeng outlining the rapid pace of change in advanced #kidneycancer#renalcellcarcinoma. A brief tweetorial. Combination approaches, IOIO and IO/VEGF have changed the paradigm for patients...1/4
https://t.co/d5XWYO4brc
For the first time, the @US_FDA has approved an #immunotherapy drug given after surgery for certain patients with #kidneycancer. The approval of adjuvant pembrolizumab was based on clinical testing led by Dana-Farber's Toni Choueiri, MD (@DrChoueiri): https://t.co/VxFB16NUN8