The mucopolysaccharidosis (MPS) disorders are a group of lysosomal storage diseases
caused by lysosomal enzyme deficits that lead to glycosaminoglycan accumulation, affecting …

Mucopolysaccharidoses (MPS) are a group of lysosomal storage disorders caused by a
deficiency in lysosomal enzymes catalyzing the stepwise degradation of …

Neurodegenerative diseases associated with old age such as Alzheimer's disease present
major problems for society, and they currently have no cure. The telomere protective caps at …

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 …

Viral vectors can be utilised to deliver therapeutic genes to diseased cells. Adeno‐
associated virus (AAV) is a commonly used viral vector that is favoured for its ability to infect …

Adeno-associated virus (AAV) vectors are one of the most efficient in vivo gene delivery
platforms. Over the past decade, clinical trials of AAV vector-mediated gene transfer led to …

Intracranial neoplasia is a common clinical condition in domestic companion animals,
particularly in dogs. Application of advances in standard diagnostic and therapeutic …

Immune tolerance is a vital component of immunity, as persistent activation of immune cells
causes significant tissue damage and loss of tolerance leads to autoimmunity. Likewise …

Pompe disease is a lysosomal storage disorder caused by acid-α-glucosidase (GAA)
deficiency, leading to glycogen storage. The disease manifests as a fatal cardiomyopathy in …

Globoid cell leukodystrophy (GLD), or Krabbe disease, is an inherited, neurologic disorder
that results from deficiency of a lysosomal enzyme, galactosylceramidase. Most commonly …