RNA therapeutics target translation rather than DNA, aiming to correct shared protein production errors. By enabling cells to read through premature stop codons, engineered tRNAs could restore ...
New findings reveal how the gene HNRNPU coordinates several fundamental molecular processes during the earliest stages of human brain development.
Structural nanomedicine — what helped give us the COVID vaccine — may now be the key to a potent blood cancer treatment that’s had remarkable early results.
“Our analyses show that the historical [human Rhinovirus] A genome represents an extinct lineage closely related to HRV A19 ...
How innovative technologies like AI and gene editing are advancing rare disease drug discovery and development.
FDA's Plausible Mechanism Framework streamlines individualized therapy approvals for ultra-rare diseases.
Rare diseases affect more people than cancer yet medicine looked the other way. Artificial intelligence is finally looking back ...
Scientists have many theories about how Earth's raw materials turned into living cells, but a new proposal is particularly ...
Collaboration, partnership, and clinical integration are key.
These findings could be used to prevent plant inbreeding and promote genetic diversity in future conservation efforts.
Today, the FDA issued draft guidance outlining a new regulatory pathway intended to help sponsors gain approval for highly ...
Men tend to lose the Y chromosome from their cells as they age. But because the Y bears few genes other than for male determination, it was thought this loss would not affect health. But evidence has ...
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