Hearing Loss in Adults | New England Journal of Medicine Skip to main content The New
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Progress in deciphering the genetic architecture of human sensorineural hearing
impairment (SNHI) or loss, and multidisciplinary studies of mouse models, have led to the …

Although genetic factors contribute to almost half of all cases of deafness, treatment options
for genetic deafness are limited,,,,. We developed a genome-editing approach to target a …

Most of the human genome encodes RNAs that do not code for proteins. These non-coding
RNAs (ncRNAs) may affect normal gene expression and disease progression, making them …

Next-generation sequencing (NGS) technology has expanded in the last decades with
significant improvements in the reliability, sequencing chemistry, pipeline analyses, data …

Splice-switching oligonucleotides (SSOs) are short, synthetic, antisense, modified nucleic
acids that base-pair with a pre-mRNA and disrupt the normal splicing repertoire of the …

Gene therapy focuses on genetic modification to produce therapeutic effects or treat
diseases by repairing or reconstructing genetic material, thus being expected to be the most …

Computational prediction of drug–target interactions (DTIs) has become an essential task in
the drug discovery process. It narrows down the search space for interactions by suggesting …

Because there are currently no biological treatments for hearing loss, we sought to advance
gene therapy approaches to treat genetic deafness. We focused on Usher syndrome, a …

Alternative splicing of pre-mRNA increases genetic diversity, and recent studies estimate
that most human multiexon genes are alternatively spliced. If this process is not highly …