In Part I of this Review, we introduced recent advances in gene delivery technologies and
explained how they have powered some of the current human gene therapy applications. In …

Lipids contain hydrocarbons and are the building blocks of cells. Lipids can naturally form
themselves into nano-films and nano-structures, micelles, reverse micelles, and liposomes …

Advances in the discovery of the causes of monogenic retinal disorders, combined with
technologies for the delivery of DNA to the retina, offer enormous opportunities for the …

There is a long history and a growing interest in the canine as a subject of study in
neuroscience research and in translational neurology. In the last few years, anatomical and …

Juvenile neuronal ceroid lipofuscinosis (JNCL) is a childhood neurodegenerative disease
with early-onset, severe central vision loss. Affected children develop seizures and CNS …

Recently, development of Adeno-associated virus (AAV) vectors has been focusing on
expanding the genetic diversity of vectors from existing sequences via directed evolution or …

Hereditary retinal dystrophies (HRDs) are degenerative diseases of the retina which have
marked clinical and genetic heterogeneity. Common presentations among these disorders …

After two decades of ups and downs, gene therapy has recently achieved a milestone in
treating patients with Leber's congenital amaurosis (LCA). LCA is a group of inherited …

Gene therapy is gaining momentum as a method of treating human disease. Initially
conceived as a strategy to complement defective genes in monogenic disorders, the scope …

Gene therapy is a technique that aims at the delivery of nucleic acids to cells, to obtain a
therapeutic effect. In situ gene therapy consists of the administration of the gene product to a …