Advancing our understanding of gene therapy research, one publication at a time
Forty years ago, the idea of gene therapy seemed like science fiction. Deliver a genetic blueprint to human cells using modified viruses or other molecular tools?
Zeroing in on Therapeutic Drug Targets
Scientific pioneers Lon Cardon and Karol Estrada shed new light on the decades-old question of how to identify optimal drug targets.
BioMarin’s Center of Technology empowers scientists with technology, resources, and collaboration
For a scientist, access to cutting-edge technologies, rare resources, and expert support can turn a new idea into a groundbreaking discovery.
Meet the Scientist: Christian Vettermann, Ph.D.
In his latest paper, Dr. Vettermann solves a complex problem facing hemophilia A scientists and clinicians for years.
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