Intracellular stability and efficacy of β-galactosidase fusions as a GM1 gangliosidosis ERT are significantly influenced by mouse versus human multi-enzyme complex interactions
Yang, Tianhong, Acosta, Walter L., Ayala, Jorge, Pattada, Shivakumar, Riley, Kassandra, Hampton, Rachel, Radin, David, Cramer, Carole L.
Published in Molecular genetics and metabolism (01.02.2023)
Published in Molecular genetics and metabolism (01.02.2023)
Get full text
Journal Article
RTB-lectin facilitates the distribution of enzymes across the blood-brain-barrier and correction in the MPS IIIA mouse model
Pattada, Shivakumar D., Ayala, Jorge, Acosta, Walter L., Yang, Tianhong, Riley, Kassandra, Hampton, Rachel, Hampton, Eddie J., Cramer, Carole L., Radin, David
Published in Molecular genetics and metabolism (01.02.2023)
Published in Molecular genetics and metabolism (01.02.2023)
Get full text
Journal Article
Preclinical enzyme replacement therapy with a recombinant β-galactosidase-lectin fusion for CNS delivery and treatment of GM1-gangliosidosis
Weesner, Jason A., van de Vlekkert, Diantha, Acosta, Walter L., Yang, Tianhong, Gomero, Elida M., Hu, Huimin, Radin, David, Cramer, Carole L., d'Azzo, Alessandra
Published in Molecular genetics and metabolism (01.02.2023)
Published in Molecular genetics and metabolism (01.02.2023)
Get full text
Journal Article
Correction of bone pathology in MPS I mouse model using lectin-mediated delivery
Acosta, Walter L., Hampton, Rachel, Ayala, Jorge, Yang, Tianhong, Pattada, Shivakumar, Riley, Kassandra, Hampton, Eddie J., Cubero, Maria Fernanda, Sanchez, Luis M., Cofre, Carmela Unnold, Martin, Reid, Solano, Aura D., Radin, David, Cramer, Carole L.
Published in Molecular genetics and metabolism (01.02.2023)
Published in Molecular genetics and metabolism (01.02.2023)
Get full text
Journal Article
Lectin-mediated delivery of α-L-iduronidase: A novel approach for MPS I enzyme replacement therapy
Acosta, Walter L., Ou, Li, Ayala, Jorge, Condori, Jose, Katta, Varun, Flory, Ashley, Martin, Reid, Radin, Jonathan, Whitley, Chester B., Cramer, Carole L., Radin, David N.
Published in Molecular genetics and metabolism (01.02.2016)
Published in Molecular genetics and metabolism (01.02.2016)
Get full text
Journal Article