Intravenous delivery for treatment of mucopolysaccharidosis type I: A comparison of AAV serotypes 9 and rh10

por Belur, Lalitha R., Podetz-Pedersen, Kelly M., Tran, Thuy An, Mesick, Joshua A., Singh, Nathaniel M., Riedl, Maureen, Vulchanova, Lucy, Kozarsky, Karen F., McIvor, R. Scott
Publicado 2020

Non-invasive intravenous administration of AAV9 transducing iduronate sulfatase leads to global metabolic correction and prevention of neurologic deficits in a mouse model of Hunte...

por Laoharawee, Kanut, Podetz-Pedersen, Kelly M., Nguyen, Tam T., Singh, Sajya M., Smith, Miles C., Belur, Lalitha R., Low, Walter C., Kozarsky, Karen F., McIvor, R. Scott
Publicado 2023

In vivo selection in non-human primates identifies superior AAV capsids for on-target CSF delivery to spinal cord

por Hanlon, Killian S., Cheng, Ming, De La Cruz, Demitri, Patel, Nikita, Santoscoy, Miguel C., Gong, Yi, Ng, Carrie, Nguyen, Diane M., Nammour, Josette, Clark, Sean W., Kozarsky, Karen, Maguire, Casey A.
Publicado 2023

Comparative Effectiveness of Intracerebroventricular, Intrathecal, and Intranasal Routes of AAV9 Vector Administration for Genetic Therapy of Neurologic Disease in Murine Mucopolys...

por Belur, Lalitha R., Romero, Megan, Lee, Junggu, Podetz-Pedersen, Kelly M., Nan, Zhenhong, Riedl, Maureen S., Vulchanova, Lucy, Kitto, Kelley F., Fairbanks, Carolyn A., Kozarsky, Karen F., Orchard, Paul J., Frey, William H., Low, Walter C., McIvor, R. Scott
Publicado 2021

Prevalence of Anti–Adeno-Associated Virus Serotype 8 Neutralizing Antibodies and Arylsulfatase B Cross-Reactive Immunologic Material in Mucopolysaccharidosis VI Patient Candidates...

por Ferla, Rita, Claudiani, Pamela, Savarese, Marco, Kozarsky, Karen, Parini, Rossella, Scarpa, Maurizio, Donati, Maria Alice, Sorge, Giovanni, Hopwood, John J., Parenti, Giancarlo, Fecarotta, Simona, Nigro, Vincenzo, Sivri, Hatice Serap, Van Der Ploeg, Ans, Andria, Generoso, Brunetti-Pierri, Nicola, Auricchio, Alberto
Publicado 2015