Hereditary diseases are caused by mutations in genes, and more than 7,000 rare diseases
affect over 30 million Americans. For more than 30 years, hundreds of researchers have …

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 …

Nucleic acids are used in many therapeutic modalities, including gene therapy, but their
ability to trigger host immune responses in vivo can lead to decreased safety and efficacy. In …

Gene transfer has long been a compelling yet elusive therapeutic modality. First mainly
considered for rare inherited disorders, gene therapy may open treatment opportunities for …

Retinal gene therapy has shown great promise in treating retinitis pigmentosa (RP), a
primary photoreceptor degeneration that leads to severe sight loss in young people. In the …

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 …

Until recently, there was no approved treatment for a retinal degenerative disease.
Subretinal injection of a recombinant adeno-associated virus (AAV) delivering the normal …

X-linked retinopathies represent a significant proportion of monogenic retinal disease. They
include progressive and stationary conditions, with and without syndromic features. Many …

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 …

Late-stage retinal degenerative disease involving photoreceptor loss can be treated by
optogenetic therapy, cell transplantation and retinal prostheses. These approaches aim to …