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Alessandro Prigione

@aleprigio.bsky.social
2.3K followers 1.6K following 84 posts

Stem Cell Metabolism lab. We use iPSCs and brain organoids to search for cures for rare mitochondrial neurological diseases. Professor at the Medical Faculty of Heinrich Heine University, Düsseldorf. Editor-in-Chief of Stem Cell Research journal. He/him.

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Reposted by Alessandro Prigione
www.MitoWorld.org @mitoworld.bsky.social · 01/07/2026
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
mitoworld.org
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...
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Alessandro Prigione @aleprigio.bsky.social · 01/07/2026
Grateful to @mitoworld.bsky.social for highlighting our work and the insightful interview!
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Alessandro Prigione @aleprigio.bsky.social · 04/06/2026
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.
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Reposted by Alessandro Prigione
www.MitoWorld.org @mitoworld.bsky.social · 04/06/2026
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
mitoworld.org
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...
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Reposted by Alessandro Prigione
Emily R. Trunnell, Ph.D. @ertrunnell.bsky.social · 02/06/2026
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
Figure 1 from the linked paper, "Readout methods in organoid applications for drug discovery
Schematic representation of human organoids, derived from either ESCs or iPSCs. ESCs are extracted from a blastocyst, whereas iPSCs are generated from somatic cells. Both ESCs and iPSCs are able to differentiate into the three germ layers (ectoderm, mesoderm, and endoderm) and can give rise to self-organizing 3D organoids. PSC-derived organoids can be used for compound screens, including the readouts and methodologies indicated on the border of the circle."Figure 2 from the linked paper: "Implementation of PSC-derived organoids in the drug discovery process
(A) The drug development process can be divided into different phases: basic research, drug discovery (assay development, primary screen, hit validation, and lead), pre-clinical development, clinical research (Phase I, Phase IIa/b, and Phase III studies), and review and approval. There is a high attrition rate of compounds during this process due to drug failure in early clinical development. Scalable and validated human PSC-derived organoids applied in drug screens can help bridge the drug development “valley of death” by providing meaningful readouts and increasing confidence in predictive value. Broader use can overcome academic resource limits and industry hesitancy, driving improvements in standardization, automation, and translational robustness.
(B) Available readouts and methods used in combination with human PSC-derived organoids for drug discovery, summarizing their respective advantages and current limitations."
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Alessandro Prigione @aleprigio.bsky.social · 27/05/2026
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!
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Alessandro Prigione @aleprigio.bsky.social · 06/05/2026
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...
nature.com
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.
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Alessandro Prigione @aleprigio.bsky.social · 05/05/2026
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...
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Alessandro Prigione @aleprigio.bsky.social · 20/04/2026
Thanks Leo for sharing! 🙏
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Alessandro Prigione @aleprigio.bsky.social · 20/04/2026
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...
nature.com
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...
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Reposted by Alessandro Prigione
Biomedical Picture of the Day BPoD @bpodaily.bsky.social · 07/04/2026
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
bpod.org.uk
BPoD | Brain Gain
Brain Gain - Biomedical pictures for April 2026.
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Reposted by Alessandro Prigione
Vito RT Zanotelli @votti.bsky.social · 12/03/2026
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 💪
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Reposted by Alessandro Prigione
Kurianlab @kurianlab.bsky.social · 12/03/2026
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
sciencedirect.com
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…
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Alessandro Prigione @aleprigio.bsky.social · 11/03/2026
#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!
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
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Alessandro Prigione @aleprigio.bsky.social · 11/03/2026
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...
science.org
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
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Alessandro Prigione @aleprigio.bsky.social · 11/03/2026
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...
sciencedirect.com
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…
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Reposted by Alessandro Prigione
Waggoner Lab @labwaggoner.bsky.social · 11/03/2026
Pluripotent stem-cell-based screening uncovers sildenafil as a mitochondrial disease therapy @cellcellpress.bsky.social @aleprigio.bsky.social www.cell.com/cell/fulltex...
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Reposted by Alessandro Prigione
International Brain Research Organization @ibroorg.bsky.social · 15/01/2026
🌍 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: ibro.org/grant/exchange-fellowships #grant #IBROinAsiaPacific #IBROinUSCanada #IBROinAfrica #IBROinLatAm
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Reposted by Alessandro Prigione
Brian Glancy @brianglancylab.bsky.social · 04/12/2025
NCLX is a mitochondrial proton/calcium exchanger, not a sodium/calcium exchanger as previously thought www.nature.com/articles/s41...
nature.com
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...
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Reposted by Alessandro Prigione
Martin Picard @martinpicardenergy.bsky.social · 06/11/2025
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
picardlab.org
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,....
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Reposted by Alessandro Prigione
Spagnoli_Lab. @labspagnoli.bsky.social · 13/11/2025
🚨 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 ⤵️
doi.org
Spatially organized cellular communities shape functional tissue architecture in the pancreas
Spatial maps reveal dynamic epithelial-mesenchymal niches shaping pancreatic development and function.
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Reposted by Alessandro Prigione
DMM Journal @dmmjournal.bsky.social · 21/11/2025
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/...
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.
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Alessandro Prigione @aleprigio.bsky.social · 21/11/2025
Thanks for the highlight @dmmjournal.bsky.social
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Reposted by Alessandro Prigione
Jean Nakhle @jeannakhle.bsky.social · 21/11/2025
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.
nature.com
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.
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Reposted by Alessandro Prigione
RyanLab_TCD @ryanlab.bsky.social · 03/11/2025
An atlas of mitochondrial ATP synthase activity across the lifespan #mitochondria www.sciencedirect.com/science/arti...
sciencedirect.com
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…
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Alessandro Prigione @aleprigio.bsky.social · 29/10/2025
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....
doi.org
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...
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Reposted by Alessandro Prigione
Nature @nature.com · 01/09/2025
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
go.nature.com
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.
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Reposted by Alessandro Prigione
Tobias Marschall @tobiasmar.bsky.social · 23/07/2025
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.
tinyurl.com
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.
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Alessandro Prigione @aleprigio.bsky.social · 23/07/2025
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...
academic.oup.com
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
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Euromit-2026 @euromit-2026.bsky.social · 10/06/2025
🎙️ Focus Speakers – Alessandro Prigione 👀 Curious to know who will be speaking at #euromit2026 ? ✨ Today, we’re introducing A. Prigione @aleprigio.bsky.social who will be taking the stage at the congress. #euromit2026 #MitochondrialDisease #mtDNA #mitochondria #angers @ua-recherche.bsky.social
Picture of Alessandro Prigione featuring his photo and a short biography. Alessandro Prigione will be speaking at Euromit congress in 2026 in Angers, France on Wednesday, June 3rd  during the session entitled  Innovative technologies. Alessandro Prigione is a professor at the Heinrich Heine University in Düsseldorf, Germany. His group uses patient-derived iPSCs to generate cerebral cells and organoids to study how mitochondrial dysfunction leads to neurological disorders such as Leigh syndrome. He uses advanced stem cell technologies, genome editing, and high-throughput drug screenings to understand disease mechanisms and identify potential therapies.
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Alessandro Prigione @aleprigio.bsky.social · 10/06/2025
Looking forward to Euromit 2026!!
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Katsu Funai @katsufunai.bsky.social · 04/06/2025
mtDNA editing!
nature.com
A mitochondrial disease model is generated and corrected using engineered base editors in rat zygotes - Nature Biotechnology
A mitochondrial disease is modeled using engineered base editors in rats.
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Alessandro Prigione @aleprigio.bsky.social · 19/05/2025
Exciting news! Our most important work is out as preprint! We suggest sildenafil as a treatment for mitochondrial disease Leigh syndrome, from stem and organoid model to mice and off-label use. Next step is the clinical trial starting early next year! www.medrxiv.org/content/10.1...
medrxiv.org
Pluripotent stem cell-based drug discovery uncovers sildenafil as a treatment for mitochondrial disease
Mitochondrial disease encompasses inherited disorders affecting mitochondrial function. A severe and untreatable form of mitochondrial disease is Leigh syndrome (LS) causing psychomotor regression and...
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Reposted by Alessandro Prigione
Nature @nature.com · 15/05/2025
Nature research paper: Spatial transcriptomics reveals human cortical layer and area specification go.nature.com/3GUX8Tm
go.nature.com
Spatial transcriptomics reveals human cortical layer and area specification - Nature
Multiplexed error-robust fluorescence in situ hybridization (MERFISH) together with deep-learning-based nucleus segmentation enabled the construction of a highly detailed and informative spatially resolved single-cell atlas of human fetal cortical development.
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Christian Frezza @frezzalab.bsky.social · 15/05/2025
Molecular machineries shaping the mitochondrial inner membrane www.nature.com/articles/s41...
nature.com
Molecular machineries shaping the mitochondrial inner membrane - Nature Reviews Molecular Cell Biology
The inner mitochondrial membrane forms cristae, which are crucial for mitochondrial function. This Review explores the protein complexes that regulate cristae dynamics, including remodelling and fusio...
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Debby Silver @debbysilver.bsky.social · 14/05/2025
Thrilled to share our latest study out in @natureportfolio.nature.com led by the fantastically talented Jing Liu. Our study provides insight into a long standing question in biology: What molecular features make us uniquely human and how do these function? www.nature.com/articles/s41...
nature.com
A human-specific enhancer fine-tunes radial glia potency and corticogenesis - Nature
HARE5, a human accelerated region enhancer, modulates cortical development by influencing neural progenitor cell behaviour, leading to an enlarged neocortex with increased functional independence betw...
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Motori Lab @motorilab.bsky.social · 05/05/2025
I'm thrilled to be co-organizing the very first EMBO Workshop bridging mitochondria and brain research - a powerhouse of science, ideas, and amazing speakers! 🧠🔬 📅 registration closes MAY 30th, so don’t wait to snag your spot! Stay tuned - more updates about the topics and speakers coming soon! 🎙️
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Leon Smyth @leonsmyth.bsky.social · 02/05/2025
Our perspective in @natrevimmunol.nature.com on new concepts in neuroimmune interactions that have challenged the old dogma that immune privilege is a result of the isolation of the nervous and immune systems. @jonykipnis.bsky.social www.nature.com/articles/s41...
nature.com
Redefining CNS immune privilege - Nature Reviews Immunology
In this Perspective, Smyth and Kipnis reappraise the concept of immune privilege in the central nervous system. Although immune privilege was originally thought to involve isolation of the central ner...
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Cell Stem Cell @cp-cellstemcell.bsky.social · 01/05/2025
Vascular network-inspired diffusible scaffolds for engineering functional midbrain organoids
dlvr.it
Vascular network-inspired diffusible scaffolds for engineering functional midbrain organoids
Guo and colleagues developed vascular network-inspired diffusible (VID) scaffolds for engineering midbrain organoids, providing a versatile technology for organoid development and therapeutic innovation.
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Eric Topol @erictopol.bsky.social · 01/05/2025
After decades of research on stem cells and their reprogramming, we're now seeing extensive clinical trial assessment, some with very encouraging results. This is a new @science.org outstanding review of the biology and ongoing research efforts www.science.org/doi/10.1126/...
science.org
Stem cells as role models for reprogramming and repair
Stem cells are a promising source for cellular therapies across many diseases and tissues. Their inherent ability to differentiate into other cell types has been the focus of investigation over decade...
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Cedric Boeckx @cedricboeckx.bsky.social · 01/05/2025
Great study by @nataliabaumann.bsky.social @djabaudon.bsky.social & collaborators, showing how regional differences in progenitor metabolism shape brain growth during development @cp-cell.bsky.social www.cell.com/cell/abstrac...
cell.com
Regional differences in progenitor metabolism shape brain growth during development
Brain-wide birthdate mapping reveals that while hindbrain neurogenesis is transient, forebrain neurogenesis is temporally sustained through spatiotemporal regulation of progenitor mitochondrial functi...
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Silvia Cappello @silvianeuro.bsky.social · 11/04/2025
www.sciencedirect.com/science/arti...
sciencedirect.com
Cellular interplay in brain organoids: Connecting cell-autonomous and non-cell-autonomous mechanisms in neurodevelopmental disease
The field of brain organoids has experienced a period of rapid and transformative growth, enabling researchers to investigate complex human biological…
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David Pla Martin @dplamartin.bsky.social · 04/04/2025
Excited to share our latest paper in Science Advances! We finally dissected a new pathway for mitochondrial quality control upon mtDNA replication stress! Let’s start a short thread! 🧵 www.science.org/doi/10.1126/...
science.org
Retromer promotes the lysosomal turnover of mtDNA
Lysosomal uptake of mtDNA controls mitochondrial quality.
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Tatjana Kleele @tatjanakleele.bsky.social · 04/04/2025
Thrilled to announce that the registration for the EMBO workshop „Mitochondria in the Nervous System“ is now open. Join us from Sep 29th to Oct 2nd in beautiful Engelberg, Switzerland! meetings.embo.org/event/25-mit...
meetings.embo.org
Mitochondria in the Nervous System
The brain is a high energy-demanding tissue with an heterogenous cellular composition. Most of the energy consumed is used for maintenance of neuronal synaptic transmission, and neurons almost exclus…
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Alessandro Prigione @aleprigio.bsky.social · 03/04/2025
Honored to be part of this landmark forum article @cp-cellstemcell.bsky.social outlining a roadmap for genomic safety in human pluripotent stem cell therapies. Thankful to Ivana Barbaric for leadership and to all coauthors for great efforts. kwnsfk27.r.eu-west-1.awstrack.me/L0/https://a...
kwnsfk27.r.eu-west-1.awstrack.me
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Richard Sever @richardsever.bsky.social · 01/04/2025
"we have been watching with growing concern policy changes in the US...The scale of disruption to science is unprecedented...We are concerned with policies that threaten censorship & removal of public data...To our colleagues in the US, we stand with you" journals.biologists.com/jcs/article/...
journals.biologists.com
Science under siege: protecting scientific progress in turbulent times
As Editors-in-Chief of The Company of Biologists' journals, we have been watching with growing concern the policy changes in the United States of America (USA) and the challenges that these are creati...
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Alessandro Prigione @aleprigio.bsky.social · 21/03/2025
Wonderful to see this out!Thanks to all clinicians for sharing their data. By looking at the natural history of Leigh syndrome patients with mitochondrial mutations MT-ATP6 and MT-ATP8 we can learn key aspects to monitor for future clinical trials. www.neurology.org/doi/10.1212/...
neurology.org
Natural History of Patients With Mitochondrial ATPase Deficiency Due to Pathogenic Variants of MT-ATP6 and MT-ATP8 | Neurology
Background and ObjectivesThe mitochondrial DNA (mtDNA) genes MT-ATP6 and MT-ATP8 encode for subunits α and 8 (A6L) of the adenosine triphosphate synthase complex. Pathogenetic variants in MT-ATP6/8 ca...
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Euromit-2026 @euromit-2026.bsky.social · 03/02/2025
🌍Euromit 2026 is coming to Angers!🌍 ✨The international congress dedicated to mitochondrial research, will take place in France, at the Centre des Congrès of Angers. from May 31 to June 4, 2026! ✨ 📢 Stay informed by following this page and get ready for an exceptional scientific experience! 🚀🔬
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Minczuk Lab @mitogene.bsky.social · 06/03/2025
REGISTRATION OPEN for @faseborg.bsky.social Meeting on Mechanisms of Mitochondrial DNA Mutation and Repair | June 1 - 5, 2025 | Nashville, Tennessee. Apply before it is too late: events.faseb.org/event/mitoch... Please re-post!
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