Andrés F. Correa-Sánchez

This is figure 1, which shows the trajectory analyses in three of the four birth cohorts.

The age that autism is diagnosed may partly reflect underlying biological and developmental differences among individuals with autism, according to a study in Nature. go.nature.com/4gQ5gSV 🧬 🧪

Characterizing Antibodies Targeting Antisense Oligonucleotide Phosphorothioate and 2′-O-Methoxyethyl Modifications for Intracellular Trafficking and Biodistribution Studies | Nucleic Acid Therapeutics The efficacy of nucleic acid therapeutics (NATs) such as antisense oligonucleotides (ASOs) and small interfering RNAs relies on multiple stages of extra- and intracellular trafficking. Assessing uptak...

We are pleased that NATA’s work is featured on the cover of Nucleic Acid Therapeutics. The study benchmarks ModDetect antibodies for detecting PS and MOE modifications in ASOs. Congrats to our Biology team.

Cover shows stained 3D cell cultures.

Read the paper: www.liebertpub.com/doi/10.1177/...

Nuclear import increases ASO activity by by RNase H and splice switching mechanisms

We have dramatically increased splice switching *and* RNase-H ASO activity by conjugating a nuclear importer!

Published in JACS (@jacs.acspublications.org ‬)

Led by Disha Kashyap, with Tom Milne+@natahub.org ‬
@uclchemistry.bsky.social 🧬🧪 #chemsky

pubs.acs.org/doi/10.1021/...

Engineering antisense oligonucleotides for targeted mRNA degradation through lysosomal trafficking Antisense oligonucleotides (ASOs) can modulate gene expression at the mRNA level, providing the ability to tackle conventionally undruggable targets and usher in an era of personalized medicine. A key...

🚨 Preprint alert 🚨 We have developed an entirely new mechanism of action for ASO gene knockdown -> lysosomal trafficking!

Works with non-DNA ASOs (unlike RNase H mech) and higher activity than corresponding gapmers!

#chemsky 🧪 @uclchemistry.bsky.social sky.social
chemrxiv.org/engage/chemr...

This is figure 1, which shows simulated contrasting precipitation responses to deforestation at different scales (0–1,000 km) over the Amazon region.

Amazonian deforestation is making wet seasons wetter and dry seasons dryer, according to research in Nature. The analysis highlights the local and regional effects of removing trees from the tropical region. https://go.nature.com/41jCClO 🧪

Why is it so hard to rewrite a genome? Synthetic biologists have the know-how and ambition to retool whole genomes. But the hidden complexity of biological systems continues to surprise them.

Synthetic biologists discuss with Nature why it’s so hard to write a genome. “We underestimated how complex biology is.” 🧪 🧬

🌍Today is Rare Disease Day!

Here’s a reminder that with over 300 million people worldwide living with a rare disease—only 5% having approved treatments—there is still so much work to be done.

At NATA, we are committed to advancing nucleic acid therapies for rare diseases.

#RareDiseaseDay

NIH cuts billions of dollars in biomedical funding, effective immediately The move halts a large slice of money for most universities and research institutions virtually overnight, imperiling vital research in everything from cancer to heart disease.

Another assault on US competitiveness at a time when biomedicine is roaring with innovation, an own-goal in a high-stakes international tournament. Of course the real losers are American people needing medicines and cures.

www.washingtonpost.com/health/2025/...

~1.5 weeks left till the deadline for my two Engineering Biology/Synthetic Biology PhD positions!

Please share 🙏 #chemsky 🧪

Controversial COVID study that promoted unproven treatment retracted after four-year saga Paper on hydroxychloroquine led by French researcher Didier Raoult is second-most-cited study ever to be withdrawn.

Controversial COVID study that promoted unproven treatment retracted after four-year saga

Paper on hydroxychloroquine led by French researcher Didier Raoult is second-most-cited study ever to be withdrawn.

@richvn.bsky.social reports at Nature.

www.nature.com/articles/d41...

Nucleic Acid Conjugates: Unlocking Therapeutic Potential Nucleic acids have emerged as a powerful class of therapeutics. Through simple base pair complementarity, nucleic acids allow the targeting of a variety of pathologically relevant proteins and RNA molecules. However, despite the preliminary successes of nucleic acids as drugs in the clinic, limited biodistribution, inadequate delivery mechanisms, and target engagement remain key challenges in the field. A key area of research has been the chemical optimization of nucleic acid backbones to significantly enhance their “drug-like” properties. Alternatively, this review focuses on the next generation of nucleic acid chemical modifications: covalent biochemical conjugates. These conjugates are being applied to improve the delivery, functionality, and targeting. Exploiting research on heterobifunctionals, such as PROTACs, RIBOTACs, molecular glues, etc., has the potential to dramatically expand nucleic acid drug functionality and target engagement capabilities. Such next-generation chemistry-based enhancements have the potential to unlock nucleic acids as effective and versatile therapeutic agents.

We just published a review in ACS Bio & Med Chem Au on the state-of-the-art and future of covalent nucleic acid conjugates as therapeutics 🧬💊!

Led by Disha Kashyap
@acspublications.bsky.social @uclchemistry.bsky.social @oxfordchemistry.bsky.social
#chemsky 🧪
pubs.acs.org/doi/10.1021/...

Eleven clinical trials that will shape medicine in 2025 - Nature Medicine Nature Medicine asks leading researchers to name their top clinical trial for 2025, from gene therapies for prion disease and sickle-cell disease to digital tools for cancer and mental health.

Nature Medicine asks leading researchers to name their top clinical trial for 2025, from gene therapies for prion disease and sickle-cell disease to digital tools for cancer and mental health. Read what they named as their top 11 trials. 🧪

One Hundred Years of Solitude review – Gabriel García Márquez’s classic makes for startling TV beauty This 16-part series does justice to Gabriel García Márquez’s magical-realist masterwork – although the novel’s sexual politics have aged badly

I remember trying to understand this book's complexity at school 🤯. The series is very well produced. Totally Recommended. www.theguardian.com/tv-and-radio...

a man in a blue suit and white turtleneck is standing next to a car . Alt: a man in a blue suit and white turtleneck is standing next to a car screaming "NOOO!".

Every time I see someone using GAPDH as a reference gene:

Academic Journals & News
(Upadated Dec 7, 2024)

go.bsky.app/hCfhqn

Trial of Controversial Alzheimer’s Drug Halted After Disappointing Results (Gift Article) Cassava Sciences said that its drug did not significantly reduce cognitive decline in 1,900 people with mild to moderate Alzheimer’s disease.

Trial of Controversial Alzheimer’s Drug Halted After Disappointing Results
Cassava Sciences said that its drug did not significantly reduce cognitive decline in 1,900 people with mild to moderate Alzheimer’s disease.
@teddyrosenbluth.bsky.social for @nytimes.com
www.nytimes.com/2024/11/25/h...

‘A place of joy’: why scientists are joining the rush to Bluesky Researchers say the social-media platform — an alternative to X — offers more control over the content they see and the people they engage with.

‘A place of joy’: why scientists are joining the rush to Bluesky

For me, it certainly feels good to be here!

@natureportfolio.bsky.social @bsky.app

www.nature.com/articles/d41...

Givinostat - Exon skipping HOW DOES GIVINOSTAT WORK? Explained by Annemieke Aartsma-Rus.

For people interested in Duchenne and wanting to know how givinostat works explained in simple terms:
www.exonskipping.nl/whats-hot/gi...
This is based on text I provided earlier for @worldduchenne.bsky.social and Italfarmaco kindly provided unrestricted funds (i.e. I was in charge) to illustrate it

Like me, you might be frustrated at the amount of overlapping and photoshopped images in science papers that we sleuths are finding.
But what is even more frustrating is the lack of response and action from journals.
Here is a thread with some outrageous examples.
#ThisImageIsFine
/

As our community grows here, reminder that our free hands on course on all aspects of modeling neural development and disease with #assembloids and #organoids will be taking place next year 👇