Underpinning Voyager’s mission is its trademark AAV capsid-discovery platform ... promise for producing candidates with enhanced tropism for liver, eye, and other tissue types (Fig.
“The tropism of natural AAV serotypes is limited,” he says. “They also tend to infect the liver, which can cause severe side effects in some cases.” Improving AAV tropism could offer ...
Simply put, AAV consists of a protein shell ... with decreased transduction of the liver. Further, we’ve seen cross-species CNS tropism (including rodents and multiple non-human primate species ...
来自MSN11 个月
Adeno-associated virus: The gene therapy revolution faces manufacturing and safety hurdlesResearchers prefer AAV vectors due to their broad tissue tropism, high safety profile ... central nervous system (CNS), lungs, retina, liver, pancreas, kidney, and heart. Natural AAV mutants ...
RNA-based therapeutics have rapidly emerged over the past decade, offering a new class of medicines that differ significantly from conventional drugs. These therapies can be programmed to target or ...
“exhibits potent editing capabilities across various cell systems and tissues in vivo when administered via adeno-associated viral (AAV) vectors.” When targeting the PCSK9 gene in mouse liver ...
Agreement allows Andelyn to use Stanton Lab CNS capsids to perform R&D services for clients developing gene therapies.
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