Impact

Photo of the backs of elderly woman holding hands with two children walking

Take a look at some of the grants and manuscripts that demonstrate the impact of our work in the Research Institute for Children’s Health at Case Western Reserve University School of Medicine.

Presentations

Rebecca Darrah presented one of the Plenary talks at the 2022 North American Cystic Fibrosis Conference.

One of our 2022 Summer Students presented a poster at the 2022 North American Cystic Fibrosis Conference. Loss of angiotensin II type-2 receptor confers protection from hepatic steatosis and mortality in ketogenic cystic fibrosis mice

Summer Student Presents Poster at NACFC 2022

Publications


K. Schelde, J. Rosenjack, C. Sonneborn, A. Jafri, M. Kavran, S. Brumbaugh, A. Rietsch, R.J. Darrah, C.A. Hodges, C.A. Flask, T.J. Kelley, M.L. Drumm1, M. A minimally invasive bronchoscopic approach for direct delivery to murine airways and application to models of pulmonary infection. Lab Anim. 2023 Dec;57(6):611-622. PMID: 37382374.


T Wei, Y Sun, Q Cheng, S Chatterjee, Z Traylor, L T Johnson, ML Coquelin, J Wang, MJ Torres, X Lian , X Wang, Y Xiao, CA Hodges, DJ Siegwart. Lung SORT LNPs enable precise homology-directed repair mediated CRISPR/Cas genome correction in cystic fibrosis models. Nat Commun. 2023 Nov 11;14(1):7322. PMID: 37951948


WB Hannah, TGJ Derks, ML Drumm, SC Grünert, PS Kishnani, J Vissing. Glycogen storage diseases. Nat Rev Dis Primers. 2023 Sep 7;9(1):46. PMID: 37679331


E. Barbato, R. Darrah, T. J. Kelley. The circadian system in cystic fibrosis mice is regulated by histone deacetylase 6. Am J Physiol Cell Physiol. 2022 Oct 1;323(4):C1112-C1120. PMID: 36062879


McBennett, K , MacAskill, C.J., Keshock, E., Mahani, M.G., Mata, J., Towbin, A.J., Sankararaman, S., Drumm, M.L., Xin Yu, X., Ren, C.L., Nasr, S.Z., Kutney, K. and Flask, C.A. Magnetic Resonance Imaging of Cystic Fibrosis: Multi-Organ Imaging in the Age of CFTR Modulator Therapies. Journal of Cystic Fibrosis 2022 Mar;21(2):e148-e157. PMID: 34879996


Plona, K., Eastman, J. and Drumm, ML. Classifying molecular phenotypes of G6PC variants for pathogenic properties and to guide therapeutic development. Journal of Inherited Metabolic Diseases Reports, 2021.

Grants

April 2022

  • Altered Circadian Rhythm Regulation in Cystic Fibrosis, National Institutes of Health, $2,504,828
  • CWRU Therapeutics Pipeline for Genetic Disease, JobsOhio, $230,000.

May 2022

  • Editing disease-causing CFTR alleles caused by segmental duplications, Cystic Fibrosis Foundation, $336,000

July 2022

  • Hartwell postdoctoral fellowship for Glycogen Storage Disease 1A, The Hartwell Foundation, $100,000.

January 2023

  • Addressing barriers to delivery and editing for Cystic Fibrosis, Cystic Fibrosis Foundation, $3,158,363.