This federal Project Grant award, totaling $173,175.00, was provided by the National Center for Advancing Translational Sciences (NCATS) under CFDA Program 93.350. The grant supports research to understand the biological effects of ATP-binding cassette A3 (ABCA3) mutations in human alveolar epithelial type 2 cells and develop a gene therapy strategy to express ABCA3 at physiological levels. This work aims to address severe respiratory distress syndrome and pulmonary fibrosis caused by ABCA3...
This $844,386 Project Grant award from the National Heart, Lung, and Blood Institute (NHLBI), under the Cardiovascular Diseases Research (CFDA 93.837) program, supports the "Integrative Genomics of Childhood Interstitial Lung Disease" research project at Boston Children's Hospital. The key objectives are to: Define patient clusters with similar molecular profiles from 300 childhood interstitial and diffuse lung disease (CHILD) lung biopsy samples, develop a CHILD molecular...
This $1,193,262 Project Grant awarded by the National Heart, Lung, and Blood Institute (NHLBI) under the Cardiovascular Diseases Research program (CFDA 93.837) aims to advance a novel adeno-associated virus (AAV)-mediated gene therapy for the treatment of idiopathic pulmonary fibrosis (IPF). The research team at the University of California, San Francisco (UCSF) will rigorously validate the efficacy of the AAV-SGPL1 gene therapy in IPF mouse models and lung tissue samples. Specifically, they...
The National Heart, Lung, and Blood Institute (NHLBI) awarded a $1,284,920 Project Grant under the Cardiovascular Diseases Research program (CFDA 93.837) to the Arkansas Children's Research Institute (ACRI). The grant aims to analyze the pharmacokinetics and pharmacogenetics of the triple combination therapy for cystic fibrosis, as well as evaluate the relationship between drug concentrations and neuropsychological side effects. A sub-award of $243,612 was made to the University of Washington to...
This Project Grant award from the National Heart, Lung, and Blood Institute (NHLBI), under the Cardiovascular Diseases Research program (CFDA 93.837), will support an investigation of pulmonary fibrosis biology in pediatric hematopoietic cell transplant patients. The $134,123 award, made on August 15, 2024, will be used by The Regents of the University of California, San Francisco (UCSF) to leverage bronchoalveolar lavage fluid and blood samples to identify biomarkers that can predict the...
This Project Grant award, provided by the National Center for Advancing Translational Sciences (NCATS) under CFDA Program 93.350, is focused on investigating the use of triacetin as a potential treatment for propionic acidemia (PA), a rare metabolic disorder. The total award amount is $184,617 and will be used to evaluate the efficacy of triacetin in rectifying metabolic abnormalities and cellular phenotypes in human PA cells, as well as assess the metabolic effects, therapeutic efficacy, and...
The National Heart, Lung, and Blood Institute (NHLBI) has awarded a $827,753 Project Grant (CFDA 93.837 - Cardiovascular Diseases Research) to The Children's Hospital Corporation (dba Boston Children's Hospital) to develop more effective gene-mapping methods for rare polygenic disorders such as idiopathic pulmonary fibrosis (IPF). The key products and services being delivered under this 1-year grant include: Extending a pleiotropy-informed association test to create a new network model-based...
This $156,136 Project Grant award from the National Center for Advancing Translational Sciences (NCATS), CFDA 93.350, aims to develop biochemical reagents and assays to analyze the structure and function of the PAX3-FOXO1 fusion protein that drives the rare disease alveolar rhabdomyosarcoma. Specifically, the project will generate recombinant PAX3-FOXO1 constructs and conduct preliminary structural screening using innovative nuclear magnetic resonance techniques. It will also establish in...
The National Heart, Lung, and Blood Institute (NHLBI) awarded The Washington University a $740,317 Project Grant under the Cardiovascular Diseases Research program (CFDA 93.837) to conduct research on the regulation of primary and secondary alveologenesis by FGF signaling pathways. This 4-year grant (1/29/2025 - 11/30/2028) will leverage unique genetic tools, single-cell sequencing, and cell sorting techniques to identify the cellular mechanisms by which FGF signaling regulates cell...
This Project Grant award, funded by the National Heart, Lung, and Blood Institute (NHLBI) under the Cardiovascular Diseases Research program (CFDA 93.837), supports research to investigate ways to improve the synthesis and assembly of the CFTR protein in individuals with cystic fibrosis (CF) who have premature termination codon (PTC) variants. The $629,319 award to Emory University aims to identify cellular targets that can ameliorate the basic defect caused by CFTR PTC variants, which are...