Thiago Carvalho @cyrilpedia@qoto.org
I've worked on all of science, from B cells to T cells.
https://fellowsherpa.com
Joined Nov 2022
Last Thursday, the FDA voted to approve Sarepta's microdystrophin gene therapy for #Duchenne muscular #dystrophy.
https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-treatment-certain-patients-duchenne-muscular-dystrophy
This was not an easy call, made on the basis of a biomarker rather than efficacy data. I wrote about it last month for Nature Medicine.
If a slipper falls on a rooftop visible from an exhibition, is it art? #discussamongstyourselves
A great week at the soon to be extinct Gulbenkian Science Institute/IGC teaching MD PhD students of the EMERALD program.
Targeting FGF21 and GLP-1 pathways in NASH. My latest Clinical Pipeline column for Nature Medicine is online.
#drugdiscovery #drugdevelopment #metabolism #LiverDisease #FattyLiver #SciComm
George and the FT might be hearing from The Onion's legal team soon.
https://www.ft.com/content/05808970-09a0-4f86-af23-4f05db627742
Targeting FGF21 & GLP-1 pathways in NASH. My latest Clinical Pipeline column for Nature Medicine is online.
#drugdiscovery #drugdevelopment #metabolism #LiverDisease #fattyliver
There's more to gasdermin than just proptosis.
'Our study supports that differential cleavage of GSDMD can be understood as a regulatory hub controlling immunity versus tolerance in the small intestine.'
#Immunology #mucosalimmunology
"For at least two centuries, scientists have been enthralled by the “zombie” behaviors induced by mind-controlling parasites. Despite this interest, the mechanistic bases of these uncanny processes have remained mostly a mystery."
'The analyses of RANK and RANKL expression in large cohorts of breast cancer samples and functional studies in RANK+ breast cancer patient-derived xenografts (PDXs) revealed a key role for RANK in postmenopausal women with estrogen receptor negative (ER-) breast cancer.'
Ciscar et al @EmboMolMed
https://www.embopress.org/doi/full/10.15252/emmm.202216715
Primer on the clinical uses of base editing tech @PLOSBiology
"In this Essay, we highlight base editing technologies and their development from bench to bedside. Base editing, first reported in 2016, is capable of installing C•G to T•A and A•T to G•C point mutations, while largely circumventing some of the pitfalls of traditional CRISPR/Cas9 gene editing."
#MolecularBiology #GeneTherapy
https://journals.plos.org/plosbiology/article?id=10.1371/journal.pbio.3002071
The von Willebrand Trust strikes again.
At the opening of "Oubliette" in Biblioteca Camões. Largo Calhariz 17 1º andar 1200-086 Lisboa. 16.06.2023 Até 30.06.2023. Exposição Coletiva.
Portrait of the artist making last minute adjustments. Congratulations to @madparreira @anaanajottajotta @jorgenesbitt @consta_arouca on a very successful opening. If you couldn't make it last night, 'Oubliette' will be at Biblioteca Camoes in Lisbon until June 30th.
#Art #artmastodon #artgallery
Back in (very sunny) Heidelberg! Looking forward to meeting students & postdocs at the Uni and reconnecting with my former colleagues at EMBO.
Who says we don't need another heron? by @hunchbacksociety #ceramics #stoneware #art #artgallery #handmade #artmastodon
It's official - we have a business! More news soon.
Celebrating with David del Álamo in Madrid.
My Clinical Pipeline column for Nature Medicine this week examines the state of Sarepta's new gene therapy for Duchenne muscular dystrophy application at the FDA. Whatever its final efficacy turns out to be, the general strategy behind the therapy, an engineered micro-dystrophin is very clever.
It was inspired by the case of a 61 year old patient who had a deletion of almost half of his dystrophin gene and was still alive and walking. Here's that ref, from 1990:
https://www.nature.com/articles/343180a0
And here's my column:
'Using an industry-standard test, he measured the particulate matter outside the mask, then checked how much made it through the device and into the space around his nose and mouth. Percentage-wise, he told me, “it removes 99.99 … I didn’t measure how many nines; it was working so well.” On broader scales, too, the protective math plays out: Well-fitting masks can curb smoke-related hospitalizations; studies back up their importance as a firefighting mainstay.'
https://www.theatlantic.com/health/archive/2023/06/mask-wildfire-smoke-n95-effective/674325/
I've worked on all of science, from B cells to T cells.
https://fellowsherpa.com
Joined Nov 2022
