Project Grant R21HD123012
- Federal Grant Award Summary Cincinnati Children's Hospital Medical Center received a $441,375 Project Grant from the National Heart, Lung, and Blood Institute (NHLBI) under the Cardiovascular Diseases Research program (CFDA 93.837), awarded August 15, 2025, with completion targeted for July 31, 2027. The award funds research to develop and optimize a dual adeno-associated virus (AAV) gene delivery system employing intein technology to deliver full-length, functional cystic fibrosis transmembrane...
- The National Institute of Child Health and Human Development awarded Yale University $100,228 on December 1, 2026, under the Child Health and Human Development Extramural Research program (CFDA 93.865) to define mechanisms of enhancer-promoter interaction in zygotic genome activation using chromatin expansion microscopy. The project combines chromatin expansion microscopy (ChromEXM) with DNA fluorescence in situ hybridization (FISH) to trace individual gene loci at near-nucleosome resolution...
- The National Institute of Child Health and Human Development awarded $430,375 to the University of California Santa Cruz on August 1, 2026, under the Child Health and Human Development Extramural Research Projects program (CFDA 93.865) to investigate the role of RNA-modifying enzymes in regulating totipotent gene expression during early embryonic development. The research explores how the putative RNA methyltransferase METTL7A1 regulates zygotic genome activation and totipotency in mouse...
- The National Institute of Child Health and Human Development awarded $644,771 to the Research Institute at Nationwide Children's Hospital on June 1, 2026, under the Child Health and Human Development Extramural Research program (CFDA 93.865, Program R01HD119141) to identify novel genes controlling craniofacial development using forward genetics in the mouse. The research uses N-ethyl-N-nitrosourea (ENU) mutagenesis to generate heritable single base-pair substitutions in the mouse genome and...
- The National Institute of Child Health and Human Development awarded Cincinnati Children's Hospital Medical Center $445,500 on September 1, 2026, under the Child Health and Human Development Extramural Research program (CFDA 93.865) to develop and test genetic and molecular assays for predicting disease severity in Fragile X Syndrome. The research addresses the absence of early quantitative biomarkers that currently prevent individualized therapeutic strategies for FXS patients. The project...
- Federal Project Grant Award Summary The Johns Hopkins University received a $426,375 Project Grant from the National Institute of Child Health and Human Development (NICHD) under the Child Health and Human Development Extramural Research program (CFDA 93.865), awarded September 26, 2025, with a completion date of August 31, 2027. The award funds development of stable CRISPR interference (CRISPRi) and CRISPR activation (CRISPRa) systems in zebrafish utilizing phic31 attP safe harbor integration...
- The National Heart, Lung, and Blood Institute awarded Cincinnati Children's Hospital Medical Center $361,125 on April 15, 2026, under the Cardiovascular Diseases Research program (CFDA 93.837) to develop FLUME, a human fetal lung multiomics cell atlas with computational models to predict fetal origins and molecular perturbations in childhood interstitial lung diseases (CHILD). The project performs secondary analysis of existing fetal lung and CHILD datasets, leveraging high-resolution spatial...
- The National Institute of Biomedical Imaging and Bioengineering awarded University of California, Berkeley $120,394 on May 1, 2026, under the Discovery and Applied Research for Technological Innovations to Improve Human Health program (CFDA 93.286) to develop genome editing delivery vehicles that amplify editing efficiency by enabling cells to produce and distribute CRISPR-Cas9 editing enzymes to neighboring cells in vesicles. The research addresses inefficient delivery of genome editing to cell...
- The National Heart, Lung, and Blood Institute awarded the Regents of the University of Michigan $167,400 on September 1, 2026, under the Cardiovascular Diseases Research program (CFDA 93.837) to develop nanoparticle-mediated delivery of CRISPR-Cas9 gene editing for sickle cell disease treatment. The project focuses on advancing non-viral platforms for therapeutic gene editing of hematopoietic stem and progenitor cells. Current gene therapy approaches using ex vivo viral transduction or...
- The National Institute of Child Health and Human Development awarded Cincinnati Children's Hospital Medical Center $866,250 on September 9, 2025, under the Child Health and Human Development Extramural Research program (CFDA 93.865) to investigate mechanisms of drug-induced liver injury in children and develop predictive biomarkers for pediatric hepatotoxicity. The project will evaluate the hepatotoxic effects of acetaminophen, valproic acid, amoxicillin-clavulanate, cannabidiol, and isoniazid...
The National Institute of Child Health and Human Development awarded Cincinnati Children's Hospital Medical Center $445,500 on September 1, 2026, under the Child Health and Human Development Extramural Research Projects program (CFDA 93.HDR) to develop and optimize a compact CRISPR activation system using the miniature Cas12f nuclease within an adeno-associated virus (AAV) vector for modulating lung vascular enhancers. The project targets regulatory DNA elements that control cell- and tissue-specific gene expression but have been traditionally considered undruggable. The system packages both guide RNAs and nuclease within the approximately 4.7 kilobyte packaging capacity of AAV, avoiding the size constraints of commonly used Cas9 and Cas12a alternatives. The recipient will design and optimize the AAV-DenascaS12f-VPR system to modulate FOXF1 enhancer activity in vivo using FELS-GFP and FOXF1-2A-GFP reporter mouse lines to systematically evaluate how guide RNA placement and enhancer architecture influence activation efficiency and specificity. The recipient will then investigate therapeutic potential in the FOXF1-S52F alveolar capillary dysplasia heterozygous mouse model to determine whether concurrent activation of the FEL1 and FEL3 endothelial enhancers can restore functional FOXF1 transcription, enhance alveolar capillary density, and improve survival. The project establishes a compact, all-in-one CRISPR activation platform based on the miniature DenascaS12f nuclease as a broadly applicable tool for modulating regulatory DNA elements. Work is performed in Cincinnati, Ohio. The period of performance runs from September 1, 2026, through August 31, 2028.
Mod # | Description | ReasonForModification | Federal Obligation | Date |
|---|---|---|---|---|
| Not listed | $445.5k | 8/19/26 |