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�…

BACKGROUND Nearly five decades ago, visionary scientists hypothesized that genetic
modification by exogenous DNA might be an effective treatment for inherited human�…

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

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�…

Tay-Sachs disease (TSD) is an inherited neurological disorder caused by deficiency of
hexosaminidase A (HexA). Here, we describe an adeno-associated virus (AAV) gene�…

Highlights•AAV is a non-pathogenic virus, and recombinant AAV vectors have proven to be
highly efficient for gene delivery to a wide variety of cell types, tissue, and organs in small�…

Aromatic L-amino acid decarboxylase deficiency results in decreased neurotransmitter
levels and severe motor dysfunction. Twenty-six patients without head control received�…

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�…

Aromatic L-amino acid decarboxylase (AADC) deficiency is a rare genetic disorder
characterized by deficient synthesis of dopamine and serotonin. It presents in early infancy�…