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 …

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 …

In recent years, adeno-associated virus (AAV) has become the most important vector for
central nervous system (CNS) gene therapy. AAV has already shown promising results in …

Adeno-associated virus (AAV) vectors exhibit a number of properties that have made this
vector system an excellent choice for both CNS gene therapy and basic neurobiological …

Adeno-associated viral (AAV) vectors are a rapidly emerging gene therapy platform for the
treatment of neurological diseases. In preclinical studies, transgenes encoding therapeutic …

Recent advancements in gene supplementation therapy are expanding the options for the
treatment of neurological disorders. Among the available delivery vehicles, adeno …

Introduction Gene therapy provides the exciting opportunity of a curative single treatment for
devastating diseases, eradicating the need for chronic medication. Adeno-associated …

Adeno-associated virus (AAV) vectors exhibit a number of properties that have made this
vector system an excellent choice for both CNS gene therapy and basic neurobiological …

As gene therapy enters mainstream medicine, it is more important than ever to have a grasp
of exactly how to leverage it for maximum benefit. The development of new targeting …

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 …