Publicaties
Spinal subpial delivery of AAV9 enables widespread gene silencing and blocks motoneuron degeneration in ALS KU Leuven
AAV9 delivered bispecific nanobody attenuates amyloid burden in the gelsolin amyloidosis mouse model Vrije Universiteit Brussel Universiteit Gent KU Leuven
Gelsolin amyloidosis is a dominantly inherited, incurable type of amyloidosis. A single point mutation in the gelsolin gene (G654A is most common) results in the loss of a Ca2+ binding site in the second gelsolin domain. Consequently, this domain partly unfolds and exposes an otherwise buried furin cleavage site at the surface. During secretion of mutant plasma gelsolin consecutive cleavage by furin and MT1-MMP results in the production of 8 ...
AAV9 delivered bispecific nanobody attenuates amyloid burden in the gelsolin amyloidosis mouse model (vol 26, pg 1353, 2017) Vrije Universiteit Brussel KU Leuven
Superior in vivo transduction of human hepatocytes using engineered AAV3 capsid Instituut voor Tropische Geneeskunde
Adeno-associated viral (AAV) vectors are currently being tested in multiple clinical trials for liver-directed gene transfer to treat the bleeding disorders hemophilia A and B and metabolic disorders. The optimal viral capsid for transduction of human hepatocytes has been under active investigation, but results across various models are inconsistent. We tested in vivo transduction in "humanized" mice. Methods to quantitate percent AAV ...