Gene transfer using adeno-associated viral (AAV) vectors has made tremendous progress in
the last decade and has achieved cures of debilitating diseases such as hemophilia A and …

Recombinant adeno-associated viruses (AAVs) have emerged as promising gene delivery
vehicles resulting in three US Food and Drug Administration (FDA) and one European …

Nine dogs with hemophilia A were treated with adeno-associated viral (AAV) gene therapy
and followed for up to 10 years. Administration of AAV8 or AAV9 vectors expressing canine …

In the last decade, enormous progress has been made in the development of gene therapy
for hemophilia A and B. After the first encouraging results of intravenously administered …

Spinal muscular atrophy is a devastating neurodegenerative autosomal recessive disease
that results from survival of motor neuron 1 (SMN1) gene mutation or deletion. Patients with …

Congenital haemophilia A (factor VIII deficiency) and B (factor IX deficiency) are X-linked
bleeding disorders. Replacement therapy has been the cornerstone of the management of …

Gene therapy is becoming an increasingly valuable tool to treat many genetic diseases with
no or limited treatment options. This is the case for hundreds of monogenic metabolic …

Gene therapy has entered a new era after decades-long efforts, where the recombinant
adeno-associated virus (AAV) has stood out as the most potent vector for in vivo gene …

Hemophilia is an X-linked inherited bleeding disorder, resulting from defects in the F8
(hemophilia A) or F9 (hemophilia B) genes. Persons with hemophilia have bleeding …

Adeno-associated virus (AAV)-mediated gene transfer has benefited patients with inherited
diseases, such as hemophilia B, by achieving long-term expression of the therapeutic …