AAV vector immunogenicity in humans: a long journey to successful gene transfer

HC Verdera, K Kuranda, F Mingozzi - Molecular Therapy, 2020 - cell.com
Gene therapy with adeno-associated virus (AAV) vectors has demonstrated safety and long-
term efficacy in a number of trials across target organs, including eye, liver, skeletal muscle …

The mannose receptor

L Martinez-Pomares - Journal of leukocyte biology, 2012 - academic.oup.com
Review on mannose receptor as an endocytic receptor in antigen delivery that is co-
expressed with the microRNA miR-511-3p encoded within the Mrc1 gene. The MR is a …

Expression systems for therapeutic glycoprotein production

Y Durocher, M Butler - Current opinion in biotechnology, 2009 - Elsevier
There are slightly over 165 recombinant pharmaceuticals currently approved for human use.
Another 500 protein candidates are in preclinical and clinical development, about 70% of …

Influence of the mannose receptor in host immune responses

U Gazi, L Martinez-Pomares - Immunobiology, 2009 - Elsevier
Mannose receptor (MR) is a C-type lectin primarily expressed by macrophages and dendritic
cells. Its three distinct extracellular binding sites recognise a wide range of both endogenous …

Adeno-associated viruses (AAV) and host immunity–a race between the hare and the hedgehog

K Rapti, D Grimm - Frontiers in immunology, 2021 - frontiersin.org
Adeno-associated viruses (AAV) have emerged as the lead vector in clinical trials and form
the basis for several approved gene therapies for human diseases, mainly owing to their …

[HTML][HTML] Endocytosis mechanisms and the cell biology of antigen presentation

S Burgdorf, C Kurts - Current opinion in immunology, 2008 - Elsevier
Recent evidence revealed that presentation of soluble antigens is governed by the
endocytosis mechanisms that determine the intracellular routing of the endocytosed …

Immunogenicity challenges associated with subcutaneous delivery of therapeutic proteins

NL Jarvi, SV Balu-Iyer - BioDrugs, 2021 - Springer
The subcutaneous route of administration has provided convenient and non-inferior delivery
of therapeutic proteins compared to intravenous infusion, but there is potential for enhanced …

The mechanistic impact of N-glycosylation on stability, pharmacokinetics, and immunogenicity of therapeutic proteins

Q Zhou, H Qiu - Journal of pharmaceutical sciences, 2019 - Elsevier
N-glycosylation is one of major post-translational modifications in nature, and it is essential
for protein structure and function. As hydrophilic moieties of glycoproteins, N-glycans play …

Recombinant factor VIII products and inhibitor development in previously untreated boys with severe hemophilia A

T Calvez, H Chambost… - Blood, The Journal …, 2014 - ashpublications.org
Six recombinant factor VIII (rFVIII) products have been marketed worldwide. In 2013, the
Research of Determinants of Inhibitor Development (RODIN) study group reported an …

The mannose receptor: from endocytic receptor and biomarker to regulator of (meta) inflammation

HJP van der Zande, D Nitsche, L Schlautmann… - Frontiers in …, 2021 - frontiersin.org
The mannose receptor is a member of the C-type lectin (CLEC) family, which can bind and
internalize a variety of endogenous and pathogen-associated ligands. Because of these …