Despite scientific discoveries in the field of gene and cell therapy, some diseases still have
no effective treatment. Advances in genetic engineering methods have enabled the …

In recent years, adeno-associated virus (AAV) has become the most important vector for
central nervous system (CNS) gene therapy. AAV has already shown promising results in …

Owing to their small size and safety profiles, adeno-associated viruses (AAVs) have become
the vector of choice for gene therapy applications in the retina. In addition to the naturally …

Large genes including several CRISPR-Cas modules like gene activators (CRISPRa)
require dual adeno-associated viral (AAV) vectors for an efficient in vivo delivery and …

X-linked retinoschisis (XLRS) is an inherited vitreoretinal dystrophy causing visual
impairment in males starting at a young age with an estimated prevalence of 1: 5000 to 1 …

Since their discovery over 55 years ago, adeno-associated virus (AAV) vectors have
become powerful tools for experimental and therapeutic in vivo gene delivery, particularly in …

Optogenetic gene therapies offer a promising strategy for restoring vision to patients with
retinal degenerative diseases, such as retinitis pigmentosa (RP). Several clinical trials have …

Owing to the promising therapeutic effect and one-time treatment advantage, gene therapy
may completely change the management of eye diseases, especially retinal diseases …

Breast carcinoma has one of the highest incidence rates (11.7%), with significant clinical
heterogeneity. Although conventional chemotherapy and surgical resection are the current …

Introduction: The eye is an excellent target for gene therapy because of its anatomical
features. Gene therapy to treat ocular disorders relies on efficient gene delivery and …