AAV-mediated PEX1 gene augmentation improves visual function in the PEX1-Gly844Asp mouse model for mild Zellweger spectrum disorder

Authors: Catherine Argyriou, Anna Polosa, Ji Yun Song, Samy Omri, Bradford Steele, Bruno Cécyre, Devin S. McDougald, Erminia Di Pietro, Jean-François Bouchard, Jean Bennett, Joseph G. Hacia, Pierre Lachapelle, Nancy E. Braverman

Source: Molecular Therapy: Methods & Clinical Development, Vol 23, Iss , Pp 225-240 (2021)

Subject Terms: peroxisome biogenesis disorder, Zellweger spectrum disorder, PEX1, retinal gene therapy, AAV therapy, metabolic disorder gene therapy, Genetics, QH426-470, Cytology, QH573-671

File Description: electronic resource

Relation: http://www.sciencedirect.com/science/article/pii/S2329050121001376; https://doaj.org/toc/2329-0501

Access URL: https://doaj.org/article/8cbf07d636cf46afa1661d18efa2eb4c

Homodimeric Minimal Factor H: In Vivo Tracking and Extended Dosing Studies in Factor H Deficient Mice

Authors: Ola Kamala, Talat H. Malik, Thomas M. Hallam, Thomas E. Cox, Yi Yang, Falguni Vyas, Saimir Luli, Chloe Connelly, Beth Gibson, Kate Smith-Jackson, Harriet Denton, Isabel Y. Pappworth, Lei Huang, David Kavanagh, Matthew C. Pickering, Kevin J. Marchbank

Source: Frontiers in Immunology, Vol 12 (2021)

Subject Terms: C3G, complement, factor H, renal disease, mouse model, AAV therapy, Immunologic diseases. Allergy, RC581-607

File Description: electronic resource

Relation: https://www.frontiersin.org/articles/10.3389/fimmu.2021.752916/full; https://doaj.org/toc/1664-3224

Access URL: https://doaj.org/article/9cc64abec9ba49d8a6e449774701123c

This result is not displayed to guests.
Login for full access.

This result is not displayed to guests.
Login for full access.

This result is not displayed to guests.
Login for full access.