Genetics

'CRISPR-Gold' fixes Duchenne muscular dystrophy mutation in mice

Scientists at the University of California, Berkeley, have engineered a new way to deliver CRISPR-Cas9 gene-editing technology inside cells and have demonstrated in mice that the technology can repair the mutation that causes ...

Genetics

Unexpected findings uncover new understanding of gene expression

Northwestern Medicine scientists have discovered that the catalytic activity of the fly enzyme Trr and mammalian MLL3/MLL4—members of the COMPASS family of proteins central to gene expression—is not required for proper ...

Genetics

New assay leads to step toward gene therapy for deaf patients

Scientists at Oregon State University have taken an important step toward gene therapy for deaf patients by developing a way to better study a large protein essential for hearing and finding a truncated version of it.

Cardiology

Electric genes hope to fix a broken heart

Broken hearts have long influenced songsters and poets, but scientists have also been occupied by its literal meaning and are now hoping to use electric gene therapy as a revolutionary new treatment for cardiac conditions.

Genetics

Intellectual disabilities caused by protein defect

Intellectual disabilities are often caused by a mutation that damages a gene, preventing the associated protein from functioning properly. However, a mutation can also change the function of a gene. As a result, the gene ...

Immunology

New test for rare immunodeficiency

Researchers at the University of Basel have developed a test to quickly and reliably diagnose a rare and severe immune defect, hepatic veno-occlusive disease with immunodeficiency. They reported on their findings in the Journal ...

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