Publications by Grantees

The following list of publications describe research carried out with CIRM funding. Click on the publication title for a brief summary of the research and for more information about the CIRM grants that contributed to the research.

Title Year Journal
ADMET-AI Enables Interpretable Predictions of Drug-Induced Cardiotoxicity. 2025 Circulation
Derivation and Characterization of Isogenic OPA1 Mutant and Control Human Pluripotent Stem Cell Lines. 2025 Cells
A framework for N-of-1 trials of individualized gene-targeted therapies for genetic diseases. 2024 Nat Commun
A novel high-titer, bifunctional lentiviral vector for autologous hematopoietic stem cell gene therapy of sickle cell disease. 2024 Mol Ther Methods Clin Dev
A Panoramic View of Cell Population Dynamics in Mammalian Aging. 2024 bioRxiv
A therapy for suppressing canonical and noncanonical SARS-CoV-2 viral entry and an intrinsic intrapulmonary inflammatory response. 2024 Proc Natl Acad Sci U S A
AMPK Drives both Glycolytic and Oxidative Metabolism in Murine and Human T Cells During Graft-versus-host Disease. 2024 Blood Adv
An age-progressive platelet differentiation path from hematopoietic stem cells causes exacerbated thrombosis. 2024 Cell
An Endothelial Cell Is Not Simply an Endothelial Cell. 2024 Stem Cells Dev
Antigen-Specific T Cell Receptor Discovery for Treating Progressive Multifocal Leukoencephalopathy. 2024 bioRxiv
Antisense oligonucleotide therapy in an individual with KIF1A-associated neurological disorder. 2024 Nat Med
Antiviral cellular therapy for enhancing T-cell reconstitution before or after hematopoietic stem cell transplantation (ACES): a two-arm, open label phase II interventional trial of pediatric patients with risk factor assessment. 2024 Nat Commun
CAR-T cell therapy targeting surface expression of TYRP1 to treat cutaneous and rare melanoma subtypes. 2024 Nat Commun
Comparing fabrication techniques for engineered cardiac tissue. 2024 J Biomed Mater Res A
CRISPRi/a screens in human iPSC-cardiomyocytes identify glycolytic activation as a druggable target for doxorubicin-induced cardiotoxicity. 2024 Cell Stem Cell
Custom-engineered hydrogels for delivery of human iPSC-derived neurons into the injured cervical spinal cord. 2024 Biomaterials
Defining metabolic flexibility in hair follicle stem cell induced squamous cell carcinoma. 2024 Sci Adv
Disease modeling and pharmacological rescue of autosomal dominant retinitis pigmentosa associated with RHO copy number variation. 2024 Elife
Early-life stress triggers long-lasting organismal resilience and longevity via tetraspanin. 2024 Sci Adv
Engineered Protein Hydrogels as Biomimetic Cellular Scaffolds. 2024 Adv Mater
Engineering allorejection-resistant CAR-NKT cells from hematopoietic stem cells for off-the-shelf cancer immunotherapy. 2024 Mol Ther
Fatty acid oxidation in immune function. 2024 Front Immunol
Functional outcome of the anterior vaginal wall in a pelvic surgery injury rat model after treatment with stem cell-derived progenitors of smooth muscle cells. 2024 Stem Cell Res Ther
Gene editing improves endoplasmic reticulum-mitochondrial contacts and unfolded protein response in Friedreich’s ataxia iPSC-derived neurons. 2024 Front Pharmacol
Generation of ‘semi-guided’ cortical organoids with complex neural oscillations. 2024 Nat Protoc
Generation of allogeneic CAR-NKT cells from hematopoietic stem and progenitor cells using a clinically guided culture method. 2024 Nat Biotechnol
Generation of antigen-specific mature T cells from RAG1(-/-)RAG2(-/-)B2M(-/-) stem cells by engineering their microenvironment. 2024 Nat Biomed Eng
Harnessing Biomaterials to Amplify Immunity in Aged Mice through T Memory Stem Cells. 2024 ACS Nano
Increased mitochondrial mutation heteroplasmy induces aging phenotypes in pluripotent stem cells and their differentiated progeny. 2024 Aging Cell
Inhibition of Pyruvate Oxidation Diminishes Melanoma Progression. 2024 J Invest Dermatol
Lentiviral vectors for precise expression to treat X-linked lymphoproliferative disease. 2024 Mol Ther Methods Clin Dev
Locoregional delivery of IL-13Ralpha2-targeting CAR-T cells in recurrent high-grade glioma: a phase 1 trial. 2024 Nat Med
Modeling Cortical Versus Hippocampal Network Dysfunction in a Human Brain Assembloid Model of Epilepsy and Intellectual Disability. 2024 bioRxiv
Multi-lineage heart-chip models drug cardiotoxicity and enhances maturation of human stem cell-derived cardiovascular cells. 2024 Lab Chip
Netrin1 patterns the dorsal spinal cord through modulation of Bmp signaling. 2024 Cell Rep
Non-invasive prenatal testing of beta-hemoglobinopathies using next generation sequencing, in-silico sequence size selection, and haplotyping. 2024 Croat Med J
Overexpression of AMPKgamma2 increases AMPK signaling to augment human T cell metabolism and function. 2024 J Biol Chem
Respiratory exposure to agricultural dust extract promotes increased intestinal Tnfalpha expression, gut barrier dysfunction, and endotoxemia in mice. 2024 Am J Physiol Gastrointest Liver Physiol
Secondary bone marrow graft loss after third-party virus-specific T cell infusion: Case report of a rare complication. 2024 Nat Commun
Small molecule inhibition of RNA binding proteins in haematologic cancer. 2024 RNA Biol
Stem cell-derived CAR T cells show greater persistence, trafficking, and viral control compared to ex vivo transduced CAR T cells. 2024 Mol Ther
Targeting IGF2BP3 enhances antileukemic effects of menin-MLL inhibition in MLL-AF4 leukemia. 2024 Blood Adv
The Benefits of Stem Cell Biology and Tissue Engineering in Low-Earth Orbit. 2024 Stem Cells Dev
The Importance, Challenges, and Possible Solutions for Sharing Proteomics Data While Safeguarding Individuals’ Privacy. 2024 Mol Cell Proteomics
Therapeutic potential of human microglia transplantation in a chimeric model of CSF1R-related leukoencephalopathy. 2024 Neuron
Ultra-high efficiency T cell reprogramming at multiple loci with SEED-Selection. 2024 bioRxiv
Updated single cell reference atlas for the starlet anemone Nematostella vectensis. 2024 Front Zool
[Cystinosis: From the gene identification to the first gene therapy clinical trial]. 2023 Med Sci (Paris)
A bio-instructive parylene-based conformal coating suppresses thrombosis and intimal hyperplasia of implantable vascular devices. 2023 Bioact Mater
A bioactive material with dual integrin-targeting ligands regulates specific endogenous cell adhesion and promotes vascularized bone regeneration in adult and fetal bone defects. 2023 Bioact Mater
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