Rodney M. Camire, Ph.D.

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Associate Professor of Pediatrics
Department: Pediatrics
Graduate Group Affiliations

Contact information
The Children's Hospital of Philadelphia
The Colket Translational Research Bldg., Room 5018
3501 Civic Center Blvd.
Philadelphia, PA 19104-4399
Office: 215-590-9968
Fax: 215-590-3660
Lab: 215-590-3873
B.A. (Biochemistry)
Saint Anselm College, 1994.
Ph.D. (Biochemistry)
University of Vermont, 1998.
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Description of Research Expertise

We study the molecular basis of blood coagulation to better understand disorders of hemostasis and develop new therapeutic approaches.

Description of Research:

My laboratory is interested in understanding the components of the blood coagulation system, how they interface with activated cells, and how disturbances in their function lead to bleeding and thrombosis. We are also interested in developing therapeutic approaches (protein and gene-based) to mitigate these events which are major causes of morbidity and mortality worldwide. We are interested in questions related to the enzymology, biochemistry, and molecular genetics of enzyme complexes involved in blood coagulation. Numerous systems are employed to answer these questions including kinetic, biophysical, and structural approaches in addition to using in vivo models to make meaningful contributions to the field. The current areas of investigation in the laboratory include:

1. Molecular basis of procofactor activation: We are interested in understanding how FV and FVIII are preserved as inactive procofactors and defining their mechanism of activation. Our work has uncovered surprising and unexpected observations that have fundamentally shifted current thinking about FV activation and its regulation by TFPI.

2. Structural correlates of protease function-basic and translational research: We seek to better understand how processing of inactive serine protease zymogens such as FX and FIX, to their active forms contributes to the expression of binding sites critical to their function. Knowledge from these biochemical studies has been applied to translational studies, some in collaboration with companies, to develop novel protein therapeutics to treat bleeding in hemophilia, trauma, or other conditions.

3. Imaging coagulation reactions in vivo. We have taken advantage of fluorescence approaches developed for physical studies of coagulation enzyme function to develop enabling technologies that permit quantitative measurements of enzyme complex assembly and function in vivo.

4. Employ gene therapy strategies for hemophilia A/B by employing novel modifications to the protein cofactor, factor VIII or zymogen FIX. Using different bioengineering strategies we are interested in modifying FVIII or FIX with unique properties that could be useful in a gene-based approach.

Keywords: serine proteinase, recombinant protein expression, blood coagulation, macromolecular enzyme complex, hemophilia, translational research, thrombosis.

Selected Publications

Sim, X., Jarocha, D., Hayes, V., Haney, H.A., Marks, M.S., Camire, R.M., French, D.L., Poncz, M., and Gadue, P: Identifying and enriching the platelet-producing human stem cell-derived megakaryocytic using factor V uptake. Blood In Press, 2017.

Polderdijk, S.G.I., Adams, T.E., Ivanciu, L., Camire, R.M., Baglin, T.P., and Huntington, J.A: Design and characterization of an APC-specific serpin for the treatment of hemophilia. Blood 129(1): 105-113, 2017.

Nguyen, G.N., George, L.A., Siner, J.I., Davidson, R.J., Zander, C.B., Zheng, X.L., Arruda, V.R., Camire, R.M., and Sabatino, S.E: Novel factor VIII variants with a modified furin cleavage site improve the efficacy of gene therapy for hemophilia A. J. Thromb. Haemost. 15(1): 110-121, 2017.

Sanataria, S., Reglinska-Matveyev, N., Gierula, M., Camire, R.M., Crawley, J.T.B., Lane, D.A., and Ahnstrom, J: Factor V anticoagulant cofactor activity that targets the early phase of coagulation. J. Biol. Chem. In Press, 2017.

Thalji, N.K., Ivanciu, L., Davidson, R., Gimotty, P.A., Krishnaswamy, S., and Camire, R.M: A rapid pro-hemostatic approach to overcome direct oral anticoagulants. Nat. Med. 22(8): 924-932, 2016.

Siner, J.I., Samelson-Jones, B.J., Crudele, J.M., French, R.A., Lee, B.J., Zhou, S., Merricks, E., Raymer, R., Nichols, T.C., Camire, R.M., and Arruda, V.R: Circumventing burin enhances factor VIII biological activity and ameliorates bleeding phenotypes in hemophilia models. JCI Insight 1(16): e89371, 2016.

Hui, D.J., Edmonson S.C., Podsakoff G.M., Pien G.C., Ivanciu L., Camire R.M., Ertl H., Mingozzi F., High K.A., and Basner-Tschakarjan E: AAV capsid CD8+ T-cell epitopes are highly conserved across AAV serotypes. Mol. There. Methods Clin. Dev. 2: 15029, 2015.

Baroni, M., Pavani, G., Pinotti, M., Branchini, A., Bernardi, F., and Camire, R.M: Asymmetric processing of mutant factor X Arg386Cys reveals differences between intrinsic and extrinsic pathway activation. Biochim. Biophys. Acta. 1854(10 Pt A): 1351-1356, 2015.

Ivanciu, L. and Camire, R.M: Hemostatic agents of broad applicability produced by selective tuning of factor Xa zymogenicity. Blood 126(1): 94-102, 2015 Notes: Journal highlighted article with a commentary.

Sehgal, A. , Barros, S., Ivanciu, L., Cooley, B., Qin, J., Racie, T., Hettinger J., Carioto, M., Jiang, Y., Brodsky, J., Prabhala, H., Zhang, X., Attarwala, H., Hutabarat, R., Foster, D., Milstein, S., Charisse, K., Kuchimanchi, S., Maier, M.A., Nechev, L., Sorensen, B., Simon, A.R., Dargaud, Y., Negrier, C., Camire, R.M., and Akinc, A: An RNAi therapeutic targeting antithrombin to rebalance the coagulation system and promote hemostasis in hemophilia. Nat. Med. 21(5): 492-497, 2015.

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Last updated: 04/18/2017
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