Clinical translation of gene therapy has been challenging, due to limitations in current
delivery vehicles such as traditional viral vectors. Herein, we report the use of gRNA: Cas9 …

The treatment of inner ear disorders remains challenging due to the intrinsic anatomical
barriers. The majority treatments and delivery approaches for accessing inner hair cells are …

Mutations in Atp2b2, an outer hair cell (OHC) gene, cause dominant hearing loss (HL) in
humans. Using a mouse model Atp2b2Obl+, with a dominant HL mutation (Oblivion), we …

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 …

We designed a unique nanocapsule for efficient single CRISPR-Cas9 capsuling,
noninvasive brain delivery and tumor cell targeting, demonstrating an effective and safe …

Background Aging, noise, infection, and ototoxic drugs are the major causes of human
acquired sensorineural hearing loss, but treatment options are limited. CRISPR/Cas9 …

Gene therapy would benefit from the effective editing of targeted cells with CRISPR-Cas9
tools. However, it is difficult to precisely assess the editing performance in vivo because the …

Genome editing technology has emerged as a potential therapeutic tool for treating
incurable diseases. In particular, the discovery of clustered regularly interspaced short …

The use of biomaterials in delivering CRISPR/Cas9 for gene therapy in infectious diseases
holds tremendous potential. This innovative approach combines the advantages of …

Therapeutic genome editing has the potential to cure diseases by directly correcting genetic
mutations in tissues and cells. Recent progress in the clustered regularly interspaced short …