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 Porcine Model of Intervertebral Disc Injury Recapitulates Human Discogenic Pain Via Notochordal Cell Loss and Pain-Inducing Nucleus Pulposus Cell Emergence. 2026 JOR Spine
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
Allogeneic CD70-Targeted Chimeric Antigen Receptor T-Cell Therapy for Advanced Renal Cell Carcinoma: Results From the Phase I TRAVERSE Trial. 2026 J Clin Oncol
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
C1q-CD44 interactions regulate microglial phagocytosis, proliferation, and migration. 2026 J Neuroinflammation
Cell-autonomous control of CAR signaling and receptor shedding via ADAM17-mediated proteolysis. 2026 Cell
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
CRISPR/Cas strategies to enhance CAR T-cell function and persistence via metabolic reprogramming. 2026 Trends Biotechnol
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
Engineered mucus-tethering bispecific nanobodies enhance mucosal immunity against respiratory pathogens. 2026 Nat Nanotechnol
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
Injectable Thermoresponsive Microgels Encapsulate and Precondition Nucleus Pulposus Cells for Intervertebral Disc Regeneration. 2026 ACS Appl Bio Mater
Integrin Alpha 4 Inhibition Prolongs the Survival of NSG Mice Engrafted with CD19-Negative Post-CART19 Relapsed B-ALL. 2026 Exp Hematol
Lesion-remote astrocytes govern microglia-mediated white matter repair. 2026 Nature
Lipid Nanoparticles for the Delivery of CRISPR/Cas9 Machinery to Enable Site-Specific Integration of CFTR and Mutation-Agnostic Disease Rescue 2026 Advanced Functional Materials
Mapping the dialogue: Decoding alveolar stem-niche interactions. 2026 Proc Natl Acad Sci U S A
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
Restoration of the immune system with base editing and non-genotoxic conditioning in a Rag2 point-mutant mouse model. 2026 Mol Ther
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
Single-cell transcriptomics-informed induced pluripotent stem cells differentiation to tenogenic lineage. 2026 Elife
Surviving the Nucleus Pulposus Desert: Next-Generation Strategies for Intervertebral Disc Cell Therapy. 2026 JOR Spine
Syndecan-1 Promotes Alveolar Type 2 Epithelial Cell Senescence during Lung Fibrosis. 2026 bioRxiv
Virus-like particles based on plant viruses and bacteriophages: emerging strategies for the delivery of nucleic acid therapeutics. 2026 Chem Sci
Wnt signaling as a regulator of memory T cells: implications for CAR-T cell therapy. 2026 Front Immunol
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