In contrast to other diverse therapies for the X-linked bleeding disorder hemophilia that are
currently in clinical development, gene therapy holds the promise of a lasting cure with a …

Protein therapeutics have drastically changed the landscape of treatment for many diseases
by providing a regimen that is highly specific and lacks many off-target toxicities. The clinical …

A first-in-human trial of diaphragmatic gene therapy (AAV1-CMV-GAA) to treat respiratory
and neural dysfunction in early-onset Pompe disease was conducted. The primary objective …

Gene therapy strategies for congenital myopathies may require repeat administration of
adeno-associated viral (AAV) vectors due to aspects of the clinical application, such as:(i) …

At first sight the bleeding disorder hemophilia A seems to have little in common with immune
disorders, but immunology research intersects with other disciplines including hematology …

A major complication of hemophilia A therapy is the development of alloantibodies
(inhibitors) that neutralize intravenously administered coagulation factor VIII (FVIII). Immune …

The devastating neurodegenerative disease multiple sclerosis (MS) could substantially
benefit from an adeno-associated virus (AAV) immunotherapy designed to restore a robust …

Hemophilia A is the X-linked bleeding disorder caused by deficiency of coagulation factor
VIII (FVIII). To address serious complications of inhibitory antibody formation in current …

Formation of pathogenic antibodies is a major problem in replacement therapies for
inherited protein deficiencies. For example, antibodies to coagulation factors ('inhibitors') …

The development of anti-drug antibodies (ADAs) is a common cause for treatment failure
and hypersensitivity reactions for many biologics. The focus of this review is the …