Achieving sufficient delivery across the blood–brain barrier is a key challenge in the
development of drugs to treat central nervous system (CNS) disorders. This is particularly …

Recombinant adeno-associated virus (AAV) is the leading vector for gene therapy in the
retina. As non-pathogenic, non-integrating, replication deficient vector, the recombinant virus …

Over the past decade, vectors derived from adeno-associated virus (AAV) have established
themselves as a powerful tool for in vivo gene transfer, allowing long-lasting and safe …

Viral vectors are potent gene delivery platforms used for the treatment of genetic and
acquired diseases. However, just as viruses have evolved to infect cells efficiently, the …

Inherited retinal dystrophies (IRDs) are a group of rare eye diseases caused by gene
mutations that result in the degradation of cone and rod photoreceptors or the retinal …

Leber congenital amaurosis (LCA) is a severe disorder resulting in visual impairment
usually starting in the first year of life. The most frequent genetic cause of LCA is an intronic …

The potential benefits of gene therapy for neurological diseases such as Parkinson's,
Amyotrophic Lateral Sclerosis (ALS), Epilepsy, and Alzheimer's are enormous. Even a delay …

Abstract The Adeno-associated viruses (AAVs) are not associated with any diseases, and
their ability to package non-genomic DNA and to transduce different cell/tissue populations …

Nonsyndromic recessive retinal dystrophies cause severe visual impairment due to the
death of photoreceptor and retinal pigment epithelium cells. These diseases until recently …

The use of vectors based on the small parvovirus adeno-associated virus has gained
significant momentum during the past decade. Their high efficiency of transduction of …