Publications by Grantees

CIRM funding supported the following publications. Click on the publication title for a brief summary of the research and for more information:

Title Year Journal
A Method to Enrich Functional Human Paneth Cells in Induced Pluripotent Stem Cell-Derived Intestinal Organoids. 2026 Cell Mol Gastroenterol Hepatol
A modular platform for automated organoid culture and longitudinal imaging. 2026 Sci Rep
A region-delineated snRNA-seq atlas of mouse spinal cord across lifespan resolves the interaction of normative aging programs with SOD1-G93A ALS. 2026 bioRxiv
A review exploring the translational perspective of artificial intelligence in drug discovery and formulation development. 2026 Ann Pharm Fr
A single-cell multiomic analysis identifies molecular and gene-regulatory mechanisms dysregulated in developing Down syndrome neocortex. 2026 Science
Aberrant Protein S-Nitrosylation Mimics the Effect of Rare Genetic Mutations in Neurodegenerative Diseases. 2026 J Neurochem
Antimicrobial proteins regulating neuroinflammation. 2026 Ann Med
Biological pacemaker induced by focal cardiac transduction with AAV-TBX18. 2026 Mol Ther
Brinzolamide microsponge-loaded in situ gel for sustained ocular delivery: design, characterization and in vivo evaluation. 2026 Naunyn Schmiedebergs Arch Pharmacol
Clinical Outcomes of Lentiviral Vector Gene Therapy for Sickle Cell Disease. 2026 Blood Adv
Comprehensive Ocular Characteristics in Cystinosis after Hematopoietic Stem-Cell Gene Therapy Over 24 Months. 2026 Am J Ophthalmol
Concordant transcriptional and morphological remodeling revealed by in vivo Perturb-CLEAR. 2026 bioRxiv
Cystinosin regulates Na(+)/H(+) exchanger 3 trafficking and function in kidney proximal tubular cells. 2026 EMBO Rep
Development and validation of a potency assay matrix for optimized and consistent manufacture of clinical mesenchymal stem/stromal cells. 2026 Front Immunol
Dissecting gene regulatory networks governing human cortical cell fate. 2026 Nature
Dynamic Reorganization of Developmental to Adult Genome Topology Controls the Initiation and Stabilization of the Human Muscle Stem Cell State. 2026 bioRxiv
Epigenetic and Transcriptional Programs Define Osteosarcoma Subtypes and Establish Targetable Vulnerabilities. 2026 Cancer Discov
Feasibility and safety of cellular therapy for in-utero repair of myelomeningocele (CuRe Trial): a first-in-human, phase 1, single-arm study. 2026 Lancet
Functional validation of the novel KIF5A p.R17Q VUS reveals defective axonal transport in iPSC-motoneurons from a SPG10 patient. 2026 Front Genet
Genome-scale functional mapping of the mammalian whole brain with in vivo Perturb-seq. 2026 bioRxiv
Hematopoietic Stem-Cell Gene Therapy for Cystinosis. 2026 N Engl J Med
Human cardiac organoids for disease modeling and drug discovery. 2026 Trends Mol Med
In vivo multiomic Perturb-seq with enhanced nuclear gRNA capture. 2026 bioRxiv
Lesion-remote astrocytes govern microglia-mediated white matter repair. 2026 Nature
Neural stem cell delivery of oncolytic CRAd-S-pk7 immunotherapy for stage 3 ovarian cancer: Dose-regimen studies. 2026 Mol Ther Oncol
On the interpretation of astrocytic calcium signalling with graphene oxide electrodes. 2026 Nat Nanotechnol
Oxidative stress-regulatory role of miR-10b-5p in the diabetic human cornea revealed through integrated multi-omics analysis. 2026 Diabetologia
Probing neuropsychiatric disorders through in vivo CRISPR screening. 2026 Curr Opin Genet Dev
Protocol for inducing dorsal spinal sensory interneurons from mouse embryonic stem cell-derived neuromesodermal progenitors. 2026 STAR Protoc
RABEP1 regulates neutrophil migration via endosomal recycling and actin polymerization. 2026 J Leukoc Biol
Reconstructing human corticogenesis: Insights from cerebral organoids into neurodevelopment and disease modeling. 2026 Dev Cell
Redirection of sphingolipid metabolism drives cytoskeletal defects in SPLIS and reveals ROCK inhibition as therapy. 2026 J Clin Invest
Single-Cell Analysis Reveals Peripheral Helper T Cells in Rheumatoid Arthritis-Related Interstitial Lung Disease. 2026 Arthritis Rheumatol
Single-Cell Proteomics Reveals Novel Cell Phenotypes in Marfan Mouse Aneurysm. 2026 Mol Cell Proteomics
Syndecan-1 Promotes Alveolar Type 2 Epithelial Cell Senescence during Lung Fibrosis. 2026 bioRxiv
[(18)F]FDG-PET and [(18)F]MPPF-PET are brain biomarkers for the creatine transporter Slc6a8 loss of function mutation. 2025 Sci Rep
A human-specific regulatory mechanism revealed in a pre-implantation model. 2025 Nature
A Living Organoid Biobank of Crohn’s Disease Patients Reveals Distinct Clinical Correlates of Molecular Disease Subtypes. 2025 medRxiv
A micro-metabolic rewiring assay for assessing hypoxia-associated cancer metabolic heterogeneity. 2025 Bioact Mater
A Rare Hematopoietic Stem Cell-Derived Megakaryocyte Progenitor Accumulates via Enhanced Survival and Contributes to Exacerbated Thrombopoiesis Upon Aging. 2025 Aging Cell
A simple approach of nuclei isolation for single nucleus multiome sequencing. 2025 J Mol Cell Cardiol
A single-cell and spatial genomics atlas of human skin fibroblasts reveals shared disease-related fibroblast subtypes across tissues. 2025 Nat Immunol
A small molecule inhibitor of RNA-binding protein IGF2BP3 shows anti-leukemic activity. 2025 Haematologica
Aberrant cytoplasmic localization of MLH1 characterizes a cell population that seeds breast cancer recurrence. 2025 Nat Commun
Aberrant S-nitrosylation in the TCA cycle contributes to mitochondrial dysfunction, energy compromise, and synapse loss in neurodegenerative diseases. 2025 Neurotherapeutics
Accelerated 3D qCEST of the Spine in a Porcine Model Using MR Multitasking at 3T. 2025 NMR Biomed
Accelerated Tempo of Cortical Neurogenesis in Down Syndrome. 2025 bioRxiv
Acholinebio-ionicliquid-functionalized gelatin methacryloyl hydrogel for chronic wound healing 2025 European Polymer Journal
Active telomere elongation by a subclass of cancer-associated POT1 mutations. 2025 Genes Dev
Adipose tissue ageing: implications for metabolic health and lifespan. 2025 Nat Rev Endocrinol
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