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
FXYD2 marks and regulates maturity of beta cells via ion channel-mediated signal transduction. 2025 Nat Commun
Generating allogeneic CAR-NKT cells for off-the-shelf cancer immunotherapy with genetically engineered HSP cells and feeder-free differentiation culture. 2025 Nat Protoc
Generation of a set of genetically modified long QT syndrome induced pluripotent stem cell lines carrying knock-in variants rs120074178 (KCNQ1 c.569G > A; p.Arg190Gln) and rs137854600 (SCN5A c.4865G > A; p.Arg1622Gln) and isogenic control lines. 2025 Stem Cell Res
Genome editing with programmable base editors in human cells. 2025 Methods Enzymol
GFP-on mouse model for interrogation of in vivo gene editing. 2025 Nat Commun
Hematopoietic stem cell gene therapy for the treatment of X-linked agammaglobulinemia. 2025 Mol Ther Methods Clin Dev
Highly efficient in vivo hematopoietic stem cell transduction using an optimized self-complementary adeno-associated virus. 2025 Mol Ther Methods Clin Dev
HIPPIE: A Multimodal Deep Learning Model for Electrophysiological Classification of Neurons. 2025 bioRxiv
Histological signatures map anti-fibrotic factors in mouse and human lungs. 2025 Nature
Histone chaperones coupled to DNA replication and transcription control divergent chromatin elements to maintain cell fate. 2025 Genes Dev
Human embryo implantation in vitro: a standardized framework for basic research and clinical translation. 2025 Fertil Steril
Human stem cell-derived GABAergic interneuron development reveals early emergence of subtype diversity and gradual electrochemical maturation. 2025 Neuron
Identification and engineering of potent bispecific antibodies that protect against herpes simplex virus recurrent disease. 2025 Cell Rep
Identification of CD84 as a potent survival factor in acute myeloid leukemia. 2025 J Clin Invest
Identification of novel genetic mutations for the treatment prognostication of canine lymphoma. 2025 NPJ Precis Oncol
IGF2BP3 redirects glycolytic flux to promote one-carbon metabolism and RNA methylation. 2025 Cell Rep
IgG autoantibodies in bullous pemphigoid induce a pathogenic MyD88-dependent pro-inflammatory response in keratinocytes. 2025 Nat Commun
Immunotherapy-related cognitive impairment after CAR T cell therapy in mice. 2025 Cell
Implantation of engineered adipocytes suppresses tumor progression in cancer models. 2025 Nat Biotechnol
In utero lipid nanoparticle delivery achieves robust editing in hematopoietic stem cells. 2025 bioRxiv
In vivo human embryonic spinal cord atlas validates stem cell-derived human dorsal interneurons and reveals ASD spinal signatures. 2025 bioRxiv
Inactivation of Histone Chaperone HIRA Unmasks a Link Between Normal Embryonic Development of Melanoblasts and Maintenance of Adult Melanocyte Stem Cells. 2025 Aging Cell
Injectable PLGA Microscaffolds with Laser-Induced Enhanced Microporosity for Nucleus Pulposus Cell Delivery. 2025 Small
Integrated Longitudinal Transcriptomic and Proteomic Analysis of the Murine Lung Response to Influenza A Virus. 2025 Am J Respir Cell Mol Biol
Integrated single-nuclei and spatial transcriptomic profiling of human sacrococcygeal teratomas reveals heterogeneity in cellular composition and X-chromosome inactivation. 2025 bioRxiv
Integrated workflow for analysis of immune enriched spatial proteomic data with IMmuneCite. 2025 Sci Rep
Intestinal epithelial PTPN2 limits pathobiont colonization by immune-directed antimicrobial responses. 2025 Gut Microbes
Intra-amniotic antisense oligonucleotide treatment improves phenotypes in preclinical models of spinal muscular atrophy. 2025 Sci Transl Med
Intraputaminal Delivery of Adeno-Associated Virus Serotype 2-Glial Cell Line-Derived Neurotrophic Factor in Mild or Moderate Parkinson’s Disease. 2025 Mov Disord
Intravenous and intracranial GD2-CAR T cells for H3K27M(+) diffuse midline gliomas. 2025 Nature
Intravenous and oral administration of the synthetic RNA drug, TY1, reverses heart failure with preserved ejection fraction in mice. 2025 Basic Res Cardiol
Investigational New Drug-enabling studies to use genetically modified mesenchymal stromal cells in patients with critical limb ischemia. 2025 Stem Cells Transl Med
Irradiation- and busulfan-free stem cell transplantation in Fanconi anemia using an anti-CD117 antibody: a phase 1b trial. 2025 Nat Med
KIF5A downregulation in spinal muscular atrophy links axonal regeneration defects with ALS. 2025 bioRxiv
Large language model agents can use tools to perform clinical calculations. 2025 NPJ Digit Med
Lentiviral vectors for hematopoietic stem cell gene therapy restore alpha-globin expression in alpha-thalassemia red blood cells. 2025 Cell Rep Med
LGR4 is essential for maintaining beta-cell homeostasis through suppression of RANK. 2025 Mol Metab
Limbal epithelial cells detected in stage III limbal stem cell deficiency by multimodal anterior segment imaging. 2025 Am J Ophthalmol
Lineage tracing of both quiescent G0 and active Hoxb5+ LT-HSCs that actively contribute to homeostatic mouse hematopoiesis. 2025 Proc Natl Acad Sci U S A
Lineage-resolved atlas of the developing human cortex. 2025 Nature
Local Growth Hormone Facilitates Aging of the Colon Epithelial Microenvironment. 2025 Aging Cell
Long noncoding RNA BCYRN1 promotes cardioprotection by enhancing human and murine regulatory T cell dynamics. 2025 J Clin Invest
Long-term preservation of muscle function and structure by repeated administration of cardiosphere-derived cells in mdx mice. 2025 Stem Cell Reports
Long-Term Safety and Efficacy of Gene Therapy for Adenosine Deaminase Deficiency. 2025 N Engl J Med
Lysosomal proteomics reveals mechanisms of neuronal apoE4-associated lysosomal dysfunction. 2025 bioRxiv
Measurement and clinical interpretation of CRISPR off-targets. 2025 Nat Genet
MeCP2 regulates telencephalic development in human cerebral organoids. 2025 Cell Rep
Meningeal solitary fibrous tumor cell states phenocopy cerebral vascular development and homeostasis. 2025 Neuro Oncol
Menthol, a consumer product additive, adversely affects human embryonic stem cells via activation of TRPM8 and TRPA1 channels. 2025 Stem Cells Transl Med
Microglia regulate GABAergic neurogenesis in prenatal human brain through IGF1. 2025 Nature