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CONTEXT: Public and patient involvement is increasingly becoming an expectation of research funders and policy makers. Not all areas of health research are public-facing. Here, we outline an approach for building the skills and developing the…
After decades of setbacks, gene therapy (GT) is experiencing major breakthroughs. Five GTs have received US regulatory approval since 2017, and over 900 others are currently in development. Many of these GTs target rare pediatric diseases that are…
