Search Results - "Crudele, Julie M."

Cas9 immunity creates challenges for CRISPR gene editing therapies by Crudele, Julie M., Chamberlain, Jeffrey S.

“…Clustered regularly interspaced short palindromic repeats (CRISPR)-Cas9 is a genome-editing technology 1 , 2 that utilizes archaeal and bacterial Cas9…”
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Eradication of neutralizing antibodies to factor VIII in canine hemophilia A after liver gene therapy by Finn, Jonathan D., Ozelo, Margareth C., Sabatino, Denise E., Franck, Helen W.G., Merricks, Elizabeth P., Crudele, Julie M., Zhou, Shangzhen, Kazazian, Haig H., Lillicrap, David, Nichols, Timothy C., Arruda, Valder R.

“…Inhibitory antibodies to factor VIII (FVIII) are a major complication in the treatment of hemophilia A, affecting approximately 20% to 30% of patients. Current…”
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AAV-based gene therapies for the muscular dystrophies by Crudele, Julie M, Chamberlain, Jeffrey S

“…Abstract Muscular dystrophy (MD) is a group of progressive genetic diseases affecting the musculature that are characterized by inflammatory infiltrates,…”
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AAV liver expression of FIX-Padua prevents and eradicates FIX inhibitor without increasing thrombogenicity in hemophilia B dogs and mice by Crudele, Julie M., Finn, Jonathan D., Siner, Joshua I., Martin, Nicholas B., Niemeyer, Glenn P., Zhou, Shangzhen, Mingozzi, Federico, Lothrop, Clinton D., Arruda, Valder R.

“…Emerging successful clinical data on gene therapy using adeno-associated viral (AAV) vector for hemophilia B (HB) showed that the risk of cellular immune…”
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Comparison of dystrophin expression following gene editing and gene replacement in an aged preclinical DMD animal model by Bengtsson, Niclas E., Crudele, Julie M., Klaiman, Jordan M., Halbert, Christine L., Hauschka, Stephen D., Chamberlain, Jeffrey S.

“…Gene editing has shown promise for correcting or bypassing dystrophin mutations in Duchenne muscular dystrophy (DMD). However, preclinical studies have focused…”
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Assessment of systemic AAV-microdystrophin gene therapy in the GRMD model of Duchenne muscular dystrophy by Birch, Sharla M, Lawlor, Michael W, Conlon, Thomas J, Guo, Lee-Jae, Crudele, Julie M, Hawkins, Eleanor C, Nghiem, Peter P, Ahn, Mihye, Meng, Hui, Beatka, Margaret J, Fickau, Brittany A, Prieto, Juan C, Styner, Martin A, Struharik, Michael J, Shanks, Courtney, Brown, Kristy J, Golebiowski, Diane, Bettis, Amanda K, Balog-Alvarez, Cynthia J, Clement, Nathalie, Coleman, Kirsten E, Corti, Manuela, Pan, Xiufang, Hauschka, Stephen D, Gonzalez, J Patrick, Morris, Carl A, Schneider, Joel S, Duan, Dongsheng, Chamberlain, Jeffrey S, Byrne, Barry J, Kornegay, Joe N

“…Duchenne muscular dystrophy (DMD) is a progressive muscle wasting disease caused by the absence of dystrophin, a membrane-stabilizing protein encoded by the…”
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Circumventing furin enhances factor VIII biological activity and ameliorates bleeding phenotypes in hemophilia models by Siner, Joshua I, Samelson-Jones, Benjamin J, Crudele, Julie M, French, Robert A, Lee, Benjamin J, Zhou, Shanzhen, Merricks, Elizabeth, Raymer, Robin, Nichols, Timothy C, Camire, Rodney M, Arruda, Valder R

“…Processing by the proprotein convertase furin is believed to be critical for the biological activity of multiple proteins involved in hemostasis, including…”
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The Role of Transgene Levels in Immune Tolerance Induction after AAV Gene Therapy in Hemophilia B Canine Models by Samelson-Jones, Ben J., French, Robert Andrew, Crudele, Julie M., Nichols, Timothy C., Arruda, Valder R

“…Recent success in AAV liver gene therapy for hemophilia A (HA) and hemophilia B (HB) has demonstrated that the lower the vector dose, the better the safety…”
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Unexpected Role of PACE/Furin Cleavage Site in FVIII Biology: Implications for Hemophilia a Therapy by Siner, Joshua I, Crudele, Julie M, Connolly, Courtney T, Zhou, Shangzhen, Merricks, Elizabeth P., Nichols, Timothy C., Camire, Rodney M., Arruda, Valder R.

“…The paired basic amino acid cleaving enzyme (PACE)/Furin is a protein convertase system that plays a vital role in several biological processes, including…”
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Tolerance Induction To FIX Padua With AAV Liver Gene Transfer In Inhibitor-Prone Hemophilia B Dogs by Crudele, Julie M, Finn, Jonathan D, Martin, Nicholas B, Siner, Joshua I, Chen, Yifeng, Zhou, Shangzhen, Niemeyer, Glenn, Lothrop, Clinton D., Mingozzi, Federico, High, Katherine A., Arruda, Valder R.

“…Emerging data from early phase clinical studies of AAV gene therapy for hemophilia B (HB) (factor IX [FIX] deficiency) show sustained expression of therapeutic…”
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Insights Into the Mechanism of Zymogen Protein C Protection Against Cancer Progression by Crudele, Julie M, Van Sluis, Geerte L., Margaritis, Paris, Siner, Joshua I, Sliozberg, Michael, Maurer, Jenna, Faella, Armida, Zhou, Shangzhen, High, Katherine A., Esmon, Charles T, Spek, C. Arnold, Arruda, Valder R

“…Abstract 3350 Cancer is frequently associated with activation of coagulation, and a procoagulant state facilitates tumor metastasis. Recent studies have…”
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Adeno-associated viral vector-driven expression of coagulation proteins for treatment of hemophilias and cancer by Crudele, Julie M

“…Treatment of hemophilia, which involves infusion of the missing clotting factor, is often hindered by the development of neutralizing antibodies to the…”
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