Hereditary diseases are caused by mutations in genes, and more than 7,000 rare diseases
affect over 30 million Americans. For more than 30 years, hundreds of researchers have …

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 …

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 …

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 …

Various binding modes of tunable metal organic frameworks (MOFs) and functional
DNAzymes (Dzs) synergistically catalyze the emergence of abundant functional …

The human genetic code encrypted in thousands of genes holds the secret for synthesis of
proteins that drive all biological processes necessary for normal life and death. Though the …

Retinal gene therapy has shown great promise in treating retinitis pigmentosa (RP), a
primary photoreceptor degeneration that leads to severe sight loss in young people. In the …

Prime editor (PE), a new genome editing tool, can generate all 12 possible base-to-base
conversions, insertion, and deletion of short fragment DNA. PE has the potential to correct …

Retinitis Pigmentosa (RP) is a hereditary retinopathy that affects about 2.5 million people
worldwide. It is characterized with progressive loss of rods and cones and causes severe …

The choriocapillaris is the innermost structure of the choroid that directly nourishes the
retinal pigment epithelium and photoreceptors. This article provides an overview of its …