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While the actual clinical application of gene therapies still faces challenges 304 such as potential toxicity, immunogenicity, and delivery efficiency, the approval of these biologics for clinical trials highlights the growing recognition and validation of AAV-mediated gene therapy as a promising and innovative treatment approach
McQuillan DJ, et al
Trial data suggest that 8 mg and 12 mg weekly are among the most effective studied maintenance ranges, but future regulatory guidance will determine how it may eventually be prescribed
Garber, K
Using quantitative trait locus mapping of the genomes of a genetically diverse mouse population, combined with fecal metagenomics and lipidomics, a recent study reported the identification of ornithine lipid (OL) as another potential immunomodulatory effector molecule produced by A
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