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Targeting STMN2 for neuroprotection and neuromuscular recovery in Spinal Muscular Atrophy: evidence from in vitro and in vivo SMA models.
Cell Mol Life Sci
December 2024
Department of Pathophysiology and Transplantation, Dino Ferrari Center, University of Milan, Milan, Italy.
The development of ground-breaking Survival Motor Neuron (SMN) replacement strategies has revolutionized the field of Spinal Muscular Atrophy (SMA) research. However, the limitations of these therapies have now become evident, highlighting the need for the development of complementary targets beyond SMN replacement. To address these challenges, here we explored, in in vitro and in vivo disease models, Stathmin-2 (STMN2), a neuronal microtubule regulator implicated in neurodegenerative diseases like Amyotrophic Lateral Sclerosis (ALS), as a novel SMN-independent target for SMA therapy.
View Article and Find Full Text PDFHealing the Cornea: Exploring the Therapeutic Solutions Offered by MSCs and MSC-derived EVs.
Prog Retin Eye Res
December 2024
Regenerative Medicine Institute, School of Medicine, University of Galway, Galway, Ireland; CURAM Centre for Research in Medical Devices, University of Galway, Galway, Ireland. Electronic address:
Affecting a large proportion of the population worldwide, corneal disorders constitute a concerning health hazard associated to compromised eyesight or blindness for most severe cases. In the last decades, mesenchymal stem/stromal cells (MSCs) demonstrated promising abilities in improving symptoms associated to corneal diseases or alleviating these affections, especially through their anti-inflammatory, immunomodulatory and pro-regenerative properties. More recently, MSC therapeutic potential was shown to be mediated by the molecules they release, and particularly by their extracellular vesicles (EVs; MSC-EVs).
View Article and Find Full Text PDFDevelopmental interplay between transcriptional alterations and a targetable cytokine signaling dependency in pediatric ETO2::GLIS2 leukemia.
Mol Cancer
September 2024
Commissariat À L'Energie Atomique Et Aux Energies Alternatives (CEA), Université Paris Cité, Institut National de La Santé Et de La Recherche Médicale (INSERM), Stabilité Génétique Cellules Souches Et Radiations, Fontenay-Aux-Roses, F-92260, France.
Article Synopsis
- The study investigates the role of the ETO2::GLIS2 fusion oncogene in pediatric acute myeloid leukemia (AML), highlighting its connection to worse outcomes in patients.
- The researchers developed models using lentiviral transduction and CRISPR-Cas9 to explore how ETO2::GLIS2 influences leukemia development in human fetal versus post-natal hematopoietic stem cells.
- They found that the presence of specific human cytokines like IL3 and SCF is crucial for leukemogenesis, suggesting that a combination treatment targeting MEK and BCL2 could effectively reduce leukemia progression.
Pluripotent stem cell-derived organoids: A brief history of curiosity-led discoveries.
Bioessays
December 2024
MRC Laboratory of Molecular Biology, Cambridge, UK.
Organoids are quickly becoming an accepted model for understanding human biology and disease. Pluripotent stem cells (PSC) provide a starting point for many organs and enable modeling of the embryonic development and maturation of such organs. The foundation of PSC-derived organoids can be found in elegant developmental studies demonstrating the remarkable ability of immature cells to undergo histogenesis even when taken out of the embryo context.
View Article and Find Full Text PDFExploring the potential of human intestinal organoids: Applications, challenges, and future directions.
Life Sci
September 2024
Department of Food and Drug, University of Parma, Parma, Italy. Electronic address:
Article Synopsis
- The gastrointestinal tract's unique environment leads to the rapid renewal of the intestinal epithelium, making it a focus for study using innovative models.
- Patient-derived intestinal organoids mimic human intestinal conditions and have potential applications across various research areas, including disease modeling, regenerative medicine, and toxicology.
- Despite their promise, challenges such as ethical issues, tissue collection, and variability in methods may limit their use in broader research and clinical settings.
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