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The pangenome provides a first glimpse of the scope of human genetic diversity. But its routine adoption into research and clinical practice faces several challenges.
After over a decade of experience with immune checkpoint inhibitors in oncology, more effort needs to be spent unraveling why some patients respond — and why the majority do not — and integrating knowledge about biomarkers into patient selection in trials.
Interest is growing in genome-editing tools that can insert large chunks of DNA into the genome — and avoid the double-strand breaks associated with CRISPR–Cas9 genotoxicity.
Two upcoming regulatory decisions represent a tipping point for commercial gene therapy, with implications for work on existing viral vectors and the pursuit of new ones.
The Broad Institute’s enlightened licensing approach to CRISPR–Cas9 intellectual property stands out in the otherwise regrettable spat for patent rights over the foundational technology.
For the raft of new ventures developing epigenome editors, a compelling niche may be diseases of haploinsufficiency or genome imprinting that require exquisite control of gene expression.
Advances in technology and changes to healthcare during the pandemic may finally realize the vision of patient-centric blood testing espoused by disgraced Theranos CEO Elizabeth Holmes.
Outrage over the cost of insulin is driving drug-pricing reform. Industry must do more to support patients dealing with spiraling out-of-pocket costs for biotech medicines.