Defective genes account for∼ 80% of the total of more than 7,000 diseases known to date.
Gene therapy brings the promise of a one-time treatment option that will fix the errors in …

In the past decade, recombinant vectors based on a non-pathogenic parvovirus, the adeno-
associated virus (AAV), have taken center stage as a gene delivery vehicle for the potential …

Genetic intervention is increasingly being explored as a therapeutic option for debilitating
disorders of the central nervous system. The safety and efficacy of gene therapies rely upon …

The emergence of genetic engineering at the beginning of the 1970′ s opened the era of
biomedical technologies, which aims to improve human health using genetic manipulation …

Background—Hennekam lymphangiectasia–lymphedema syndrome (Online Mendelian
Inheritance in Man 235510) is a rare autosomal recessive disease, which is associated with …

Over the past 5 years, our laboratory has systematically developed a structure-guided library
approach to evolve new adeno-associated virus (AAV) capsids with altered tissue tropism …

More than one hundred naturally occurring variants of adeno-associated virus (AAV) have
been identified, and this library has been further expanded by an array of techniques for …

In this report, we describe the development of a modified adeno‐associated virus (AAV)
capsid and promoter for transduction of retinal ON‐bipolar cells. The bipolar cells, which are …

Recent progress in gene transfer technology enables the delivery of genes precisely to the
application-relevant cell type ex vivo on cultivated primary cells or in vivo on local or …

Gene transfer vectors derived from the adeno-associated virus (AAV) have recently received
increasing attention due to substantial therapeutic benefit in several clinical trials …