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 …

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 …

In retinitis pigmentosa, loss of cone photoreceptors leads to blindness, and preservation of
cone function is a major therapeutic goal. However, cone loss is thought to occur as a …

A resurgence of interest and investment in the field of gene therapy, driven in large part by
advances in viral vector technology, has recently culminated in United States Food and Drug …

Adeno-associated virus (AAV) vectors have emerged as a gene-delivery platform with
demonstrated safety and efficacy in a handful of clinical trials for monogenic disorders …

Reliable genome editing via Clustered Regularly Interspaced Short Palindromic Repeat
(CRISPR)/Cas9 may provide a means to correct inherited diseases in patients. As proof of …

Adeno-associated viral vectors (AAVs) have become popular for gene therapy, given their
many advantages, including their reduced inflammatory profile compared with that of other …

Retinitis pigmentosa refers to a diverse group of hereditary diseases that lead to incurable
blindness, affecting two million people worldwide. As a common pathology, rod …

Targeting genes to specific neuronal or glial cell types is valuable for both understanding
and repairing brain circuits. Adeno-associated viruses (AAVs) are frequently used for gene …

The light-gated cation channel channelrhodopsin-2 (ChR2) has rapidly become an
important tool in neuroscience, and its use is being considered in therapeutic interventions …