Whoa 🤯 in Slow-MoIn San Francisco, researchers are preparing the first human trial of #in-utero #GeneTherapy, starting with a rare lysosomal storage disorder.
archive.md/ap00C
archive.md/ap00C
Levka"Gene therapies to fix failing hearts gain steam after years in the doldrums
Treatments aim to bulk up failing hearts to restore their vigour — but can the field move on from past controversy?
The first clinical trial aimed at growing new heart-muscle cells is now under way, and companies are developing at least four other regenerative gene therapies for heart conditions.
'These are the first-in-human studies to take regeneration into the clinic,' says Andrew Baker, a gene-therapy researcher at the University of Edinburgh, UK, who is unaffiliated with the efforts. 'It’s a very exciting time.'
But the excitement is tempered with caution. The mammalian heart is bad at repairing itself and 'is notoriously unwieldy when it comes to efforts to try to get it to regenerate', says Sean Wu, a cardiologist at Stanford University in California. Some scientists are unconvinced that the data underpinning the existing clinical trial show true regeneration in the form of cell division. And efforts to regenerate the heart are still haunted by a controversy that led to the retraction of at least a dozen papers and the closure of a high-profile laboratory.
Developing a gene therapy in this field will be difficult, says Antonio Abbate, a cardiologist at the University of Virginia in Charlottesville. But 'we have to do research because, eventually, we’ll get it right'"
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