Gene therapy has been successfully used to treat a number of diseases, including immune deficiencies, hereditary blindness, ...
Background/aims Posterior capsule opacification (PCO) is the most frequent long-term complication after cataract surgery, caused by proliferation of residual lens epithelial cells (LECs). Metformin, a ...
The advent of near-haploid human cells, containing one copy of most chromosomes instead of two, has had a profound impact on the field of genetic screening and drug target identification. With no ...
Inborn errors of metabolism (IEMs) are rare, devastating disorders caused by pathogenic variants in genes encoding key metabolic enzymes, with the liver playing a critical role in over 140 IEMs. Liver ...
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