Brain malformation is often caused by genetic mutations. Deciphering the mutations in
patient-derived tissues has identified potential causative factors of the diseases. To validate …

The CRISPR–Cas9 system is a powerful tool for genome editing, which can potentially lead
to new therapies for genetic diseases. To date, various viral and non-viral delivery systems …

A recent study published in Science Translational Medicine by Xiao et al. reported the use of
mini dCas13X-based adenosine base editor (mxABE) to correct a mutant transcript and …

Transient delivery of CRISPR‐based genome editing effectors is important to reduce off‐
target effects and immune responses. Recently extracellular vesicles (EVs) have been …

CRISPR/Cas9 genome editing has gained rapidly increasing attentions in recent years,
however, the translation of this biotechnology into therapy has been hindered by efficient …

Genome editing using CRISPR/Cas9 has attracted considerable attention for the treatment
of genetic disorders and viral infections. Co-delivery of Cas9 mRNA and single guide (sg) …

CRISPR-based genome editing holds immense promise for enabling genetic therapies for
countless diseases, but delivery technology—rife with limitations—remains a major …

Nanoparticle-based CRISPR/Cas9 delivery systems hold great promise for specific and
precise treatment of genetic disorder diseases. Herein, we developed a DNA nanoflower …

Noise‐induced hearing loss (NIHL) poses a significant burden on not only the economics of
health care but also the quality of life of an individual, as we approach an unprecedented …

Guide RNAs offer programmability for CRISPR-Cas9 genome editing but also add
challenges for delivery. Chemical modification, which has been key to the success of …