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 …

Adeno-associated virus (AAV) has emerged as a pivotal delivery tool in clinical gene
therapy owing to its minimal pathogenicity and ability to establish long-term gene expression …

Gene therapy is at the cusp of a revolution for treating a large spectrum of CNS disorders by
providing a durable therapeutic protein via a single administration. Adeno-associated virus …

Mucopolysaccharidosis type IIIA is a severe degenerative disease caused by an autosomal
recessive defect of a gene encoding a lysosomal heparan-N-sulfamidase, the N …

In recent years, gene therapy has been raising hopes toward viable treatment strategies for
rare genetic diseases for which there has been almost exclusively supportive treatment. We …

Adeno-associated virus (AAV) is a member of the family Parvoviridae that has been widely
used as a vector for gene therapy because of its safety profile, its ability to transduce both …

Background Neurodevelopmental disorders, as a class of diseases, have been particularly
difficult to treat even when the underlying cause (s), such as genetic alterations, are …

The neuronal ceroid lipofuscinoses comprise a group of neurodegenerative lysosomal
storage disorders caused by mutations in at least 13 different genes and primarily affect the …

Clinical gene therapy has made important advances over the last decade. Among
neurological diseases, severe genetic neurodegenerative conditions have been the focus of …

Gene therapy has strong potential for treating a variety of genetic disorders, as
demonstrated in recent clinical trials. There is unfortunately no scarcity of disease targets …