Thanks Leo for sharing! 🙏

Accelerating Leigh syndrome drug discovery through deep learning screening in brain organoids - Nature Communications Leigh syndrome is a severe, currently untreatable mitochondrial neurological disorder. Menacho, Okawa et al. developed a deep-learning drug screening pipeline in brain organoids, identifying azole com...

Super excited to share our latest work! In collaboration with Antonio del Sol, we show that brain organoids coupled with deep learning screening allow uncovering new repurposable drugs for the incurable mitochondrial disease Leigh syndrome.
www.nature.com/articles/s41...

BPoD | Brain Gain Brain Gain - Biomedical pictures for April 2026.

Brain organoids used to screen drug candidates for genetic mitochondrial disease Leigh Syndrome identifies utility of sildenafil (Viagra)

📷 Annika Zink, Dao-Fu Dai, Annika Wittich et al @aleprigio.bsky.social
@hhu.de in @cp-cell.bsky.social

➡️ bpod.org.uk/archive/2026... +
@rooph.bsky.social

Thank you very much for the kind words!

Really impressive study showing an actual successful end-to-end rare disease example how to use IPSC + repurposable drug screening followed up by increasing complex diseases models (and functional analyses) to find a drug that is successfully tested in patients.

Congratulations 💪

Thank you so much Manu!1🙏

Thank you very much! We are grateful and thankful for your highly professional handling!

Thanks so much Andrew!! I did not realize that. This is so funny! I guess this is the advantage of having almost 100 coauthors..

Pluripotent stem-cell-based screening uncovers sildenafil as a mitochondrial disease therapy Mitochondrial disease encompasses inherited disorders affecting mitochondrial function. A severe and untreatable form of mitochondrial disease is Leig…

www.sciencedirect.com/science/arti...

Check this tour de force by @aleprigio.bsky.social and collegues

Brilliant paper. Showing the true potential of hiPSC-based disease modeling studies to tackle real-world therapies

Image shows a mitochondrion. In the lower left, fragmented lines represent impaired energy production and cellular stress in diseased cells. From the upper right, a blue light wave symbolizes the sildenafil intervention displacing the visual noise associated with mitochondrial dysfunction

#NotTheCover We proposed this image for our paper
out now in CELL @cellpress.bsky.social

It depicts sildenafil treatment like a wave displacing the visual noise associated with mitochondrial dysfunction.

Fantastic work from Emma Vidal at DrawImpacts!

Interesting! I will pass the info to the clinicians!

Viagra compound may hold promise for treating fatal genetic disease Animal studies and preliminary human data support the safety of sildenafil in Leigh syndrome, but the jury’s out on whether it’s effective

Insightful commentary in @science.org on our paper out now in @cellpress.bsky.social. Thank you very much to Catherine Offord for writing this piece and to @vgamalab.bsky.social for the supportive words!

www.science.org/content/arti...

Pluripotent stem-cell-based screening uncovers sildenafil as a mitochondrial disease therapy Mitochondrial disease encompasses inherited disorders affecting mitochondrial function. A severe and untreatable form of mitochondrial disease is Leig…

Our collaborative work is out now in CELL @cellpress.bsky.social

We sugggest sildenafil as a repurposable drug for mitochondrial disease.

A clinical trial is planned to start later this year.

Thanks to collaborators, funders, and patient organizations!

www.sciencedirect.com/science/arti...

Pluripotent stem-cell-based screening uncovers sildenafil as a mitochondrial disease therapy @cellcellpress.bsky.social @aleprigio.bsky.social
www.cell.com/cell/fulltex...

🌍 Applications are open! The IBRO Exchange Fellowships give early career #neuroscientists to conduct lab visits with several expenses covered during the exchange.

🗓 Apply by 15 Apr: https://ibro.org/grant/exchange-fellowships/

#grant #IBROinAsiaPacific #IBROinUSCanada #IBROinAfrica #IBROinLatAm

Structure and mechanism of the mitochondrial calcium transporter NCLX - Nature Cryogenic electron microscopy structures and functional analyses reveal that NCLX functions as a H+/Ca2+ rather than a Na+/Ca2+ exchanger, and uncover its transport mechanism with implications for the...

NCLX is a mitochondrial proton/calcium exchanger, not a sodium/calcium exchanger as previously thought
www.nature.com/articles/s41...

MiSBIE Study The Mitochondrial Stress, Brain Imaging, and Epigenetics (MiSBIE) study is a NIH-funded research platform allowing investigators to ask deeply interdisciplinary questions around brain-body processes,....

Interested in energy-based thinking around health?
Interested in mitochondria and body-mind processes?

Join us for the 2025 Mitochondrial Stress, Brain Imaging, and Epigenetics (MiSBIE) Symposium on December 12th.

Poster abstract submission open.

www.picardlab.org/misbie

Spatially organized cellular communities shape functional tissue architecture in the pancreas Spatial maps reveal dynamic epithelial-mesenchymal niches shaping pancreatic development and function.

🚨 Check out our new paper is now online in its final version!!!
🎉 👏 Congratulations @alejotorrescano.bsky.social who led the study
+ past & current team @labspagnoli.bsky.social
+ collaborators #VigilanteLab @kingslsm.bsky.social

➡️ doi.org/10.1126/scia...

short 🧵 below & more acknowledgments ⤵️

Diagram illustrating altered neurodevelopment in Huntington’s disease (HD) and proposed early interventions. The top row shows a healthy brain trajectory: wild-type huntingtin (wtHTT) supports normal development from a healthy developing brain to a healthy adult brain. The bottom row shows HD trajectory: presence of mutant huntingtin (mHTT) leads to an aberrant developing brain and later regional brain atrophy. Below, two intervention strategies to preserve developmental compensation are shown: Cell-autonomous approaches target stress response mechanisms (CHCHD2, DRP1, ATM, HSF1, other factors) to preserve mitochondrial health; Non-cell-autonomous approaches support intercellular communication, with compensation by cerebellum, healthy cells, and glial cells.

A new #DMMPerspective from Wenqing Xu & @aleprigio.bsky.social explores increasing evidence of #neurodevelopmental aspects in #HuntingtonsDisease & early interventions that might lead to prevention or delay of the disease pathology

journals.biologists.com/dmm/article/...

Thanks for the highlight @dmmjournal.bsky.social

The metabolic engine of cognition: microglia–neuron interactions in health, ageing and disease - Nature Metabolism This Review highlights how metabolic interactions between microglia and neurons shape brain health, and how their disruption in ageing and disease contributes to cognitive decline.

Out now in @natmetabolism.nature.com 🚨

This Review highlights how metabolic interactions between microglia and neurons shape brain health, and how their disruption in ageing as well as in metabolic, neuroinflammatory and neurodegenerative diseases contributes to cognitive decline.

Congrats Christian and team!!

An atlas of mitochondrial ATP synthase activity across the lifespan Mitochondrial dysfunction and declining energy production are hallmarks of aging, yet we lack a comprehensive systems-level view of ATP synthase (Comp…

An atlas of mitochondrial ATP synthase activity across the lifespan #mitochondria
www.sciencedirect.com/science/arti...

Brain organoid models of Huntington's disease shift the focus towards neurodevelopment Summary: Increasing evidence indicates neurodevelopmental aspects in Huntington's disease. Early interventions promoting healthy brain development and mitochondrial fitness might lead to prevention or...

Our latest perspective is out in Dis Model Mech (@biologist.bsky.social)!
We discuss how brain organoids highlight the impact of Huntington's disease on brain development. We suggest takling mitochondrial fitness early on a as a potential treatment strategy.

doi.org/10.1242/dmm....

Fantastic discovery! Congratulations!

Cool! Congratulations Carmelo and team!!

Cells are swapping their mitochondria. What does this mean for our health? Researchers are studying why the energy factories are moving between cells and whether the process can be harnessed to treat cancer and other diseases.

Researchers are studying why mitochondria are moving between cells and whether the process can be harnessed to treat cancer and other diseases

go.nature.com/4fYoEwG

Complex genetic variation in nearly complete human genomes - Nature Using sequencing and haplotype-resolved assembly of 65 diverse human genomes, complex regions including the major histocompatibility complex and centromeres are analysed.

Two papers in today's issue of @nature.com ‬: 1) we assemble 65 genomes to near completion, including centromeres and the MHC. tinyurl.com/3huhax6w. 2) we sequence 1,019 genomes from the 1kGP with long reads, revealing SVs down to low allele frequencies tinyurl.com/wbx3we9x.

Neuronal branching in stem cell models of mitochondrial and neurological diseases Abstract. Neuronal branching, the extension and arborization of neurites, is critical for establishing and maintaining functional neural circuits. Emerging

Our latest review is out in STEM CELLS!
We discuss the role of mitochondria in neuronal branching and how iPSC models in 2D and 3D can be used dissect the impact of neuronal branching defects in mitochondrial disorders.

academic.oup.com/stmcls/advan...