Repurposing drugs could point to treatment for #LeighSyndrome, a fatal #MitochondrialDisease, says #NatureCommunications @natureportfolio.nature.com paper by Drs. Del Sol & @aleprigio.bsky.social : mitoworld.org/repurposing-... #UMDF #mitoscientists #mitochondria #rarediseases #AIdrugdiscovery

Repurposing Drugs to Treat Leigh Syndrome - MitoWorld

Leigh syndrome (LS) is an untreatable mitochondrial disorder that leads to psychomotor regression and early death. Recently, an international research team, led by Antonio Del Sol and Alessandro Prigi...

mitoworld.org

Grateful to @mitoworld.bsky.social for highlighting our work and the insightful interview!

www.MitoWorld.org @mitoworld.bsky.social · last mo.

Repurposing drugs could point to treatment for #LeighSyndrome, a fatal #MitochondrialDisease, says #NatureCommunications @natureportfolio.nature.com paper by Drs. Del Sol & @aleprigio.bsky.social : mitoworld.org/repurposing-... #UMDF #mitoscientists #mitochondria #rarediseases #AIdrugdiscovery

Thank you very much @mitoworld.bsky.social for highlighting our work and the upcoming trial! We plan to start recruiting individuals with MT-ATP6 variants later this year.

www.MitoWorld.org @mitoworld.bsky.social · 2mo ago

A patient‑derived iPSC screen by Prigione identified sildenafil as a potential therapy for #LeighSyndrome, a devastating #mitochondrialdisease. @cp-cell.bsky.social Now recruiting for clinical trials! mitoworld.org/using-ips-ce... #UMDF #mitoscientists #mitochondria #SynLeigh @ema.europa.eu

A patient‑derived iPSC screen by Prigione identified sildenafil as a potential therapy for #LeighSyndrome, a devastating #mitochondrialdisease. @cp-cell.bsky.social Now recruiting for clinical trials! mitoworld.org/using-ips-ce... #UMDF #mitoscientists #mitochondria #SynLeigh @ema.europa.eu

Using iPS Cells to Find Therapies for Mitochondrial Diseases - MitoWorld

A screen of iPS cells recently identified sildenafil as a potential treatment for Leigh syndrome (LS). Moreover, further testing of the drug showed marked improvement in animal models and human patien...

mitoworld.org

In this new review Wittich et al., including @aleprigio.bsky.social, discuss human stem cell–derived #organoids as increasingly powerful 3D models for #drugdiscovery, improving disease modeling & drug screening by better capturing human tissue structure & function. 🧪 #NAMs #biotech

Fantastic work from @andrearossilab.bsky.social Rossi lab on the development of @cleanfinder.bsky.social for nuclear and mitochondrial genome editing application. Greatful that my lab have contributed!

RossiLab@andrearossilab.bsky.social · 3mo ago

Excited to share that our work on CleanFinder is published in Trends in Biotechnology! 🎉 Analyzing complex allele mixtures across editing modalities and sequencing platforms is a major bottleneck. We developed CleanFinder to solve this. Read it here: www.sciencedirect.com/science/arti...

In the midst of bad news in the word, it was amazing and humbling to see our recent discovery of sildenafil in Leigh syndrome in @cp-cell.bsky.social highlighted among five inspiring uplifting stories in @nature.com www.nature.com/articles/d41...

The news is not all bad: five inspiring science stories to lift your mood

Species recovery, cancer-preventing vaccines and progress in developing renewable-energy sources are just some of the positive developments that have happened this year so far.

nature.com

Our spotlight article is out in Cell Metabolism highlighting the exciting publication by Du et al in Cell showing the effectiveness of mitochondrial transplantation @cellpress.bsky.social authors.elsevier.com/a/1n2ge5WXUl...

Thanks Leo for sharing! 🙏

Kurianlab@kurianlab.bsky.social · 4mo ago

www.nature.com/articles/s41... Check this from @aleprigio.bsky.social

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...

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...

nature.com

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

BPoD | Brain Gain

Brain Gain - Biomedical pictures for April 2026.

bpod.org.uk

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 💪

Alessandro Prigione@aleprigio.bsky.social · 5mo ago

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...

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

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…

sciencedirect.com

#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!

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...

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

science.org

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

Mitochondrial disease encompasses inherited disorders affecting mitochondrial function. A severe and untreatable form of mitochondrial disease is Leig…

sciencedirect.com

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

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

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...

nature.com

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

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,....

picardlab.org

🚨 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 ⤵️

Spatially organized cellular communities shape functional tissue architecture in the pancreas

Spatial maps reveal dynamic epithelial-mesenchymal niches shaping pancreatic development and function.

doi.org

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

DMM Journal@dmmjournal.bsky.social · 9mo ago

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/...

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.

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.

nature.com

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

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…

sciencedirect.com

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....

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...

doi.org

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

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.

go.nature.com

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.

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.

tinyurl.com

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...

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

academic.oup.com