Recombinant Adeno-Associated Viral Integration and Genotoxicity: Insights from Animal Models von Chandler, Randy J, Sands, Mark S, Venditti, Charles P

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Messenger RNA therapy as an option for treating metabolic disorders von Chandler, Randy J.

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Evaluating the state of the science for adeno-associated virus integration: An integrated perspective von Sabatino, Denise E., Bushman, Frederic D., Chandler, Randy J., Crystal, Ronald G., Davidson, Beverly L., Dolmetsch, Ricardo, Eggan, Kevin C., Gao, Guangping, Gil-Farina, Irene, Kay, Mark A., McCarty, Douglas M., Montini, Eugenio, Ndu, Adora, Yuan, Jing

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Determining recombinant AAV capsid extracellular and intracellular biodistribution by dual radioisotope labeling von Chandler, Randy J.

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Gene Therapy for Methylmalonic Acidemia: Past, Present, and Future von Chandler, Randy J, Venditti, Charles P

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Gene therapy for organic acidemias: Lessons learned from methylmalonic and propionic acidemia von Chandler, Randy J., Venditti, Charles P.

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Systemic Treatment for Postnatal, Juvenile, and Runted Adult Mice by Retrobulbar Sinus Injection von Romero, Darwin, Chandler, Randy J

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Combined methylmalonic acidemia and homocystinuria, cblC type. I. Clinical presentations, diagnosis and management von Carrillo-Carrasco, Nuria, Chandler, Randy J., Venditti, Charles P.

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From Puppies to adults: In vivo editing of hepatocytes in a canine model of glycogen storage disease type Ia von Chandler, Randy J.

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Systemic Messenger RNA Therapy as a Treatment for Methylmalonic Acidemia von An, Ding, Schneller, Jessica L., Frassetto, Andrea, Liang, Shi, Zhu, Xuling, Park, Ji-Sun, Theisen, Matt, Hong, Sue-Jean, Zhou, Jenny, Rajendran, Raj, Levy, Becca, Howell, Rebecca, Besin, Gilles, Presnyak, Vladimir, Sabnis, Staci, Murphy-Benenato, Kerry E., Kumarasinghe, E. Sathyajith, Salerno, Timothy, Mihai, Cosmin, Lukacs, Christine M., Chandler, Randy J., Guey, Lin T., Venditti, Charles P., Martini, Paolo G.V.

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Vector design influences hepatic genotoxicity after adeno-associated virus gene therapy von Chandler, Randy J, LaFave, Matthew C, Varshney, Gaurav K, Trivedi, Niraj S, Carrillo-Carrasco, Nuria, Senac, Julien S, Wu, Weiwei, Hoffmann, Victoria, Elkahloun, Abdel G, Burgess, Shawn M, Venditti, Charles P

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Promoterless, Nuclease‐Free Genome Editing Confers a Growth Advantage for Corrected Hepatocytes in Mice With Methylmalonic Acidemia von Chandler, Randy J., Venturoni, Leah E., Liao, Jing, Hubbard, Brandon T., Schneller, Jessica L., Hoffmann, Victoria, Gordo, Susana, Zang, Shengwen, Ko, Chih‐Wei, Chau, Nelson, Chiang, Kyle, Kay, Mark A., Barzel, Adi, Venditti, Charles P.

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Growth advantage of corrected hepatocytes in a juvenile model of methylmalonic acidemia following liver directed adeno-associated viral mediated nuclease-free genome editing von Venturoni, Leah E., Chandler, Randy J., Liao, Jing, Hoffmann, Victoria, Ramesh, Nikhil, Gordo, Susana, Chau, Nelson, Venditti, Charles P.

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Enhanced Efficacy and Increased Long-Term Toxicity of CNS-Directed, AAV-Based Combination Therapy for Krabbe Disease von Li, Yedda, Miller, Christopher A., Shea, Lauren K., Jiang, Xuntian, Guzman, Miguel A., Chandler, Randy J., Ramakrishnan, Sai M., Smith, Stephanie N., Venditti, Charles P., Vogler, Carole A., Ory, Daniel S., Ley, Timothy J., Sands, Mark S.

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Successfully Navigating Food and Drug Administration Orphan Drug and Rare Pediatric Disease Designations for AAV9-hPCCA Gene Therapy: The National Institutes of Health Platform Vec... von Lomash, Richa Madan, Shchelochkov, Oleg, Chandler, Randy J, Venditti, Charles P, Pariser, Anne R, Ottinger, Elizabeth A

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Mitochondrial dysfunction in mut methylmalonic acidemia von Chandler, Randy J, Zerfas, Patricia M, Shanske, Sara, Sloan, Jennifer, Hoffmann, Victoria, DiMauro, Salvatore, Venditti, Charles P

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Targeting proximal tubule mitochondrial dysfunction attenuates the renal disease of methylmalonic acidemia von Manoli, Irini, Sysol, Justin R., Li, Lingli, Houillier, Pascal, Garone, Caterina, Wang, Cindy, Zerfas, Patricia M., Cusmano-Ozog, Kristina, Young, Sarah, Trivedi, Niraj S., Cheng, Jun, Sloan, Jennifer L., Chandler, Randy J., Abu-Asab, Mones, Tsokos, Maria, Elkahloun, Abdel G., Rosen, Seymour, Enns, Gregory M., Berry, Gerard T., Hoffmann, Victoria, DiMauro, Salvatore, Schnermann, Jurgen, Venditti, Charles P.

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Systemic AAV9 gene therapy improves the lifespan of mice with Niemann-Pick disease, type C1 von Chandler, Randy J, Williams, Ian M, Gibson, Alana L, Davidson, Cristin D, Incao, Arturo A, Hubbard, Brandon T, Porter, Forbes D, Pavan, William J, Venditti, Charles P

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Genotoxicity in Mice Following AAV Gene Delivery: A Safety Concern for Human Gene Therapy? von Chandler, Randy J, LaFave, Matthew C, Varshney, Gaurav K, Burgess, Shawn M, Venditti, Charles P

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Long-term Rescue of a Lethal Murine Model of Methylmalonic Acidemia Using Adeno associated Viral Gene Therapy von Chandler, Randy J, Venditti, Charles P

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