Recent medical advances have exploited the ability to address a given disease at the
underlying level of transcription and translation. These treatment paradigms utilize nucleic …

Gene therapy with adeno-associated virus (AAV) vectors has demonstrated safety and long-
term efficacy in a number of trials across target organs, including eye, liver, skeletal muscle …

Ocular delivery of lipid nanoparticle (LNPs) packaged mRNA can enable efficient gene
delivery and editing. We generated LNP variants through the inclusion of positively charged …

Gene therapy involves introduction of exogenous genetic materials into the cells in order to
correct a specific pathological condition. However, efficient delivery of the genetic materials …

The field of gene therapy has made significant strides over the last several decades toward
the treatment of previously untreatable genetic disease. Gene therapy techniques have …

Genetic diseases seriously threaten human health and have always been one of the
refractory conditions facing humanity. Currently, gene therapy drugs such as siRNA, shRNA …

Gene therapy promises to revolutionize biomedicine and personalized medicine by
modulating or compensating the expression of abnormal genes. The biggest obstacle for …

Gene therapy using recombinant adeno‐associated virus (rAAV) vectors to treat blinding
retinal dystrophies has become clinical reality. Therapeutically impactful targeting of …

Gene therapy is an evolving field in medicine and ophthalmology. Its success and
application depends on several factors that are specific to the disease to treat. For now, we …

Inherited retinal diseases (IRDs) comprise a heterogeneous group of genetic disorders
affecting the retina. Caused by mutations in over 300 genes, IRDs result in visual impairment …