Introduction of exogenous genes into target cells to overcome various tumor diseases
caused by genetic defects or abnormalities and gene therapy, a new treatment method …

Retinitis pigmentosa (RP) poses a significant threat to eye health worldwide, with
prevalence rates of 1 in 5000 worldwide. This genetically diverse retinopathy is …

Retinitis pigmentosa (RP) is a complex spectrum of inherited retinal diseases marked by the
gradual loss of photoreceptor cells, ultimately leading to blindness. Among these, mutations …

Retinitis pigmentosa (RP) is a progressive inherited retinal dystrophy, characterized by the
degeneration of photoreceptors, presenting as a rod-cone dystrophy. Approximately 20-30 …

Purpose To assess the safety and feasibility of direct vitrectomy-sparing subretinal injection
for gene delivery in a large animal model. Methods The experimental Liběchov minipigs …

Cytidine base editors (CBEs) hold significant potential in genetic disease treatment and in
breeding superior traits into animals. However, their large protein sizes limit their delivery by …

The recent years have witnessed the rapid advancement of clustered regularly interspaced
short palindromic repeats/associated protein 9 (CRISPR/Cas9)-mediated gene editing in …

Current therapeutic options for hearing loss rely on hearing aids, ossiculoplasty or cochlear
implants. These devices have limitations, particularly in noisy acoustic environments …

Induced pluripotent stem cells (iPSCs) are a powerful tool for biomedical research, but their
production presents challenges and safety concerns. Yamanaka and Takahashi …

Despite compelling empirical evidence demonstrating its efficacy, gene therapies for usher
syndrome (USH) are not yet available for the patient's usage. This scoping review assessed …