Recombinant adeno-associated virus (AAV) vectors have been, or are currently in use, in
332 phase I/II/III clinical trials in a number of human diseases, and in some cases …

Hearing loss stands as the most prevalent sensory deficit among humans, posing a
significant global health challenge. Projections indicate that by 2050, approximately 10% of …

Hearing loss is the most common sensory deficit experienced by humans and represents
one of the largest chronic health conditions worldwide. It is expected that around 10% of the …

Hyperlipidemia is a medical condition characterized by high levels of lipids in the blood. It is
often associated with an increased risk of cardiovascular diseases such as heart attacks and …

Over the past decade, in vivo gene replacement therapy has significantly advanced,
resulting in market approval of numerous therapeutics predominantly relying on adeno …

Diabetic retinopathy (DR) is considered a primarily microvascular complication of diabetes.
Müller glia cells are at the centre of the retinal neurovascular unit and play a critical role in …

Duchenne muscular dystrophy (DMD) is a progressive X-linked disease caused by
mutations in the DMD gene that prevent the expression of a functional dystrophin protein …

Genetic disorders of the central nervous system (CNS) comprise a significant portion of
disability in both children and adults. Several preclinical animal models have shown …

Atrial fibrillation (AF) is the most prevalent cardiac arrhythmia, progressive in nature, and
known to have a negative impact on mortality, morbidity, and quality of life. Patients requiring …

The dosing interval for effective recombinant adeno-associated virus (rAAV)-mediated gene
therapy of cystic fibrosis lung disease remains unknown. Here, we assessed the durability of …