Exon-skipping antisense oligonucleotides for cystic fibrosis therapy
Significance Cystic fibrosis (CF) is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which can lead to respiratory failure. To date,
Significance Cystic fibrosis (CF) is caused by mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, which can lead to respiratory failure. To date,
Cancer is driven by multiple genetic alterations, from mutations to aneuploidy. Aneuploidy is the presence of an abnormal number of chromosomes in cancer cells. Although
Abstract Genetic variants account for approximately half the cases of congenital and early‐onset deafness. Methods and technologies for viral delivery of genes into the inner
Bottom Line: Among patients with lung cancer from Latin America, genomic and ancestry analyses revealed that Native American ancestry was associated with increased mutations in
About half a million people, most of them children living in Africa, are killed each year by malaria1. Management of malaria, particularly that caused by
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