Zobrazeno 1 - 3
of 3
pro vyhledávání: '"Marina Sokolski‐Papkov"'
AAV gene replacement therapy for treating MPS IIIC: Facilitating bystander effects via EV‐mRNA cargo
Autor:
Tierra A. Bobo, Michael Robinson, Christopher Tofade, Marina Sokolski‐Papkov, Peter Nichols, Stephen Vorobiov, Haiyan Fu
Publikováno v:
Journal of Extracellular Vesicles, Vol 13, Iss 7, Pp n/a-n/a (2024)
Abstract MPS IIIC is a lysosomal storage disease caused by mutations in heparan‐α‐glucosaminide N‐acetyltransferase (HGSNAT), for which no treatment is available. Because HGSNAT is a trans‐lysosomal‐membrane protein, gene therapy for MPS I
Externí odkaz:
https://doaj.org/article/f3b1a7a5a82f4abd9c55af1cc1da434d
Facilitate by-stander effects by EV-mRNA cargo in AAV gene replacement therapy for treating MPS IIIC
Publikováno v:
Molecular Genetics and Metabolism. 135:S23-S24
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_________::ce8aea570583c13fa00a9beb61b2d835
https://doi.org/10.1016/j.ymgme.2021.11.042
https://doi.org/10.1016/j.ymgme.2021.11.042
Autor:
Marina Sokolski-Papkov, Solomiia Borova, Daniel Nahm, Thomas Lorson, Rainer Jordan, Michael M. Lübtow, Alexander V. Kabanov, Malik Salman Haider, Erik Wegener, Robert Luxenhofer
Publikováno v:
Biomaterials. 178
Poly(2-oxazoline)s have been investigated for decades as biomaterials. Pioneering early work suggested that hydrophilic poly(2-oxazoline)s are comparable to poly(ethylene glycol) regarding their potential as biomaterials, but the ready commercial ava
Externí odkaz:
https://explore.openaire.eu/search/publication?articleId=doi_dedup___::9c308057239047675939a7f51aef509a
https://pubmed.ncbi.nlm.nih.gov/29945064
https://pubmed.ncbi.nlm.nih.gov/29945064