Adeno-associated virus (AAV) based viral vectors are widely used in human gene therapy
and form the basis of approved treatments for several genetic diseases. Immune responses …

As the clinical experience in adeno-associated viral (AAV) vector-based gene therapies is
expanding, the necessity to better understand and control the host immune responses is …

Adeno-associated virus (AAV) vectors are used for correcting multiple genetic disorders.
Although the goal is to achieve lifelong correction with a single vector administration, the …

Dorsal root ganglion (DRG) toxicity has been consistently reported as a potential safety
concern after delivery of adeno-associated viruses (AAVs) containing gene-replacement …

Adeno-associated virus (AAV) vectors represent a novel tool for the delivery of genetic
therapeutics and enable the treatment of a wide range of diseases. Success of this new …

Gene therapy is a promising therapeutic approach for treating life-threatening disorders.
Despite the clinical improvements observed with gene therapy, immune responses either …

Systemic dosing of adeno-associated viral (AAV) vectors poses potential risk of adverse side
effects including complement activation triggered by anti-capsid immunity. Due to the …

Hepatitis B virus (HBV) has afflicted humankind for decades and there is still no treatment
that can clear the infection. The development of recombinant adeno-associated virus (rAAV) …

Recombinant adeno-associated virus (rAAV) vectors have emerged as a promising tool for
gene therapy. However, the systemic administration of rAAV vectors is not without risks …

Cardiac muscle targeting is a notoriously difficult task. Although various nanoparticle (NP)
and adeno-associated viral (AAV) strategies with heart tissue tropism have been developed …