AAV9 delivered bispecific nanobody attenuates amyloid burden in the gelsolin amyloidosis mouse model
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DOI: 10.1093/hmg/ddx056
Abstract: 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… read more here.
Keywords: amyloid burden; gelsolin; bispecific nanobody; amyloidosis ... See more keywords