Hereditary degenerations of the human retina are genetically heterogeneous, with well over
100 genes implicated so far. This Seminar focuses on the subset of diseases called retinitis …

Inherited retinal dystrophies (IRDs) are a group of clinically and genetically heterogeneous
degenerative disorders. To date, mutations have been associated with IRDs in over 270 …

Background The eye is an excellent candidate for gene therapy as it is immune privileged
and much of the disease-causing genetics are well understood. Towards this goal, we …

RNA interference (RNAi) can be used to inhibit the expression of specific genes in vitro and
in vivo, thereby providing an extremely useful tool for investigating gene function. Progress …

Mutational heterogeneity represents a significant barrier to development of therapies for
many dominantly inherited diseases. For example,> 100 mutations in the rhodopsin gene …

Mutational heterogeneity represents one of the greatest barriers impeding the progress
toward the clinic of gene therapies for many dominantly inherited disorders. A general …

Autosomal dominant retinitis pigmentosa (ADRP) is frequently caused by mutations in RHO,
the gene for rod photoreceptor opsin. Earlier, a study on mice carrying mutated rhodopsin …

A wide range of retinal disorders can potentially be treated using viral vector-mediated gene
therapy. The most widely used vectors for ocular gene delivery are based on adeno …

The use of nanoparticles as carriers for the delivery of therapeutic materials to target tissues
has became popular in recent years and has demonstrated great potentials for the …

Many mutations in the human rhodopsin gene (RHO) cause autosomal dominant retinitis
pigmentosa (ADRP). Our previous studies with a P23H (proline-23 substituted by histidine) …