According to Darwin's theory, endless evolution leads to a revolution. One such example is
the Clustered Regularly Interspaced Palindromic Repeats (CRISPR)–Cas system, an …

Within less than a decade since its inception, CRISPR-Cas9-based genome editing has
been rapidly advanced to human clinical trials in multiple disease areas. Although it is highly …

Progress in deciphering the genetic architecture of human sensorineural hearing
impairment (SNHI) or loss, and multidisciplinary studies of mouse models, have led to the …

Programmable RNA editing tools enable the reversible correction of mutant transcripts,
reducing the potential risk associated with permanent genetic changes associated with the …

The development of clustered regularly interspaced short-palindromic repeat (CRISPR)-
based biotechnologies has revolutionized the life sciences and introduced new therapeutic …

Inherited and age-related retinal degeneration is the hallmark of a large group of
heterogeneous diseases and is the main cause of untreatable blindness today. Genetic …

Mutations in Atp2b2, an outer hair cell gene, cause dominant hearing loss in humans. Using
a mouse model Atp2b2 Obl/+, with a dominant hearing loss mutation (Oblivion), we show …

Adeno-associated virus (AAV) vectors have shown promising results in preclinical models,
but the genomic consequences of transduction with AAV vectors encoding CRISPR-Cas …

The discovery of clustered, regularly interspaced short palindromic repeats (CRISPR) and
their cooperation with CRISPR-associated (Cas) genes is one of the greatest advances of …

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 …