Search Results - "Erger, Kirsten E"

LHON gene therapy vector prevents visual loss and optic neuropathy induced by G11778A mutant mitochondrial DNA: biodistribution and toxicology profile by Koilkonda, Rajeshwari, Yu, Hong, Talla, Venu, Porciatti, Vittorio, Feuer, William J, Hauswirth, William W, Chiodo, Vince, Erger, Kirsten E, Boye, Sanford L, Lewin, Alfred S, Conlon, Thomas J, Renner, Lauren, Neuringer, Martha, Detrisac, Carol, Guy, John

“…To demonstrate safety and efficacy of allotopic human ND4 for treatment of a Leber's hereditary optic neuropathy (LHON) mouse model harboring the G11778A…”
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In vitro evaluation of mitochondrial dysfunction and treatment with adeno-associated virus vector in fibroblasts from Doberman Pinschers with dilated cardiomyopathy and a pyruvate dehydrogenase kinase 4 mutation by Sosa, Ivan, Estrada, Amara H, Winter, Brandy D, Erger, Kirsten E, Conlon, Thomas J

“…OBJECTIVE To compare mitochondrial oxygen consumption rate (OCR) of fibroblasts from Doberman Pinschers with and without dilated cardiomyopathy (DCM) and…”
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Identification of adeno-associated viral vectors suitable for intestinal gene delivery and modulation of experimental colitis by Polyak, Steven, Mach, Annette, Porvasnik, Stacy, Dixon, Lisa, Conlon, Thomas, Erger, Kirsten E, Acosta, Andres, Wright, Amy J, Campbell-Thompson, Martha, Zolotukhin, Irene, Wasserfall, Clive, Mah, Cathryn

“…Effective gene transfer with sustained gene expression is an important adjunct to the study of intestinal inflammation and future therapy in inflammatory bowel…”
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Gene therapy for leber congenital amaurosis caused by RPE65 mutations: safety and efficacy in 15 children and adults followed up to 3 years by Jacobson, Samuel G, Cideciyan, Artur V, Ratnakaram, Ramakrishna, Heon, Elise, Schwartz, Sharon B, Roman, Alejandro J, Peden, Marc C, Aleman, Tomas S, Boye, Sanford L, Sumaroka, Alexander, Conlon, Thomas J, Calcedo, Roberto, Pang, Ji-Jing, Erger, Kirsten E, Olivares, Melani B, Mullins, Cristina L, Swider, Malgorzata, Kaushal, Shalesh, Feuer, William J, Iannaccone, Alessandro, Fishman, Gerald A, Stone, Edwin M, Byrne, Barry J, Hauswirth, William W

“…To determine the safety and efficacy of subretinal gene therapy in the RPE65 form of Leber congenital amaurosis using recombinant adeno-associated virus 2…”
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Transfer of Therapeutic Genes into Fetal Rhesus Monkeys Using Recombinant Adeno-Associated Type I Viral Vectors by Conlon, Thomas J, Mah, Cathryn S, Pacak, Christina A, Rucker Henninger, Mary B, Erger, Kirsten E, Jorgensen, Marda L, Lee, C Chang I, Tarantal, Alice F, Byrne, Barry J

“…Neuromuscular disorders such as Pompe disease (glycogen storage disease, type II), result in early and potentially irreversible cellular damage with a very…”
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In vivo expression of human ATP:cob(I)alamin adenosyltransferase (ATR) using recombinant adeno-associated virus (rAAV) serotypes 2 and 8 by Erger, Kirsten E., Conlon, Thomas J., Leal, Nicole A., Zori, Robert, Bobik, Thomas A., Flotte, Terence R.

“…Background Methylmalonic aciduria (MMA) is an autosomal recessive disease with symptoms that include ketoacidosis, lethargy, recurrent vomiting, dehydration,…”
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An acidic oligopeptide displayed on AAV2 improves axial muscle tropism after systemic delivery by Lee, Ni-Chung, Falk, Darin J, Byrne, Barry J, Conlon, Thomas J, Clement, Nathalie, Porvasnik, Stacy, Jorgensen, Marda L, Potter, Mark, Erger, Kirsten E, Watson, Rachael, Ghivizzani, Steven C, Chiu, Hung-Chuan, Chien, Yin-Hsiu, Hwu, Wuh-Liang

“…The appropriate tropism of adeno-associated virus (AAV) vectors that are systemically injected is crucial for successful gene therapy when local injection is…”
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Next generation treatment of Pompe disease using systemic gene transfer with AAV9 by Falk, Darin, Soustek, Meghan S., Mah, Cathryn S., Cloutier, Denise A., Germain, Sean A., Kelley, Jeffry S., Erger, Kirsten E., Conlon, Thomas J., Clement, Nathalie, Byrne, Barry J.

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