Despite scientific discoveries in the field of gene and cell therapy, some diseases still have
no effective treatment. Advances in genetic engineering methods have enabled the …

Parvoviruses are a diverse family of small, non-enveloped DNA viruses that infect a wide
variety of species, tissues and cell types. For over half a century, their intriguing biology and …

Since its first identification in Scotland, over 1,000 cases of unexplained paediatric hepatitis
in children have been reported worldwide, including 278 cases in the UK. Here we report an …

The therapeutic use of adeno-associated viral vector (AAV)-mediated gene disruption using
CRISPR-Cas9 is limited by potential off-target modifications and the risk of uncontrolled …

Recombinant adeno-associated virus (rAAV) vector gene delivery systems have
demonstrated great promise in clinical trials but continue to face durability and dose-related …

Conclusions: In conclusion, MLIV. K and MLIV. A are AAV peptide display variants selected
in immunocompetent mice with improved hepatocyte tropism and transduction efficiency …

The liver is a prime target for in vivo gene therapies using recombinant adeno-associated
viral vectors. Multiple clinical trials have been undertaken for this target in the past 15 years; …

Recent clinical successes have intensified interest in using adeno-associated virus (AAV)
vectors for therapeutic gene delivery. The liver is a key clinical target, given its critical …

Primary adrenal insufficiency (PAI) occurs in 1 in 5 to 7000 adults. Leading etiologies are
autoimmune adrenalitis in adults and congenital adrenal hyperplasia (CAH) in children. Oral …

Viral vectors based on recombinant adeno-associated virus (rAAV) have become the most
widely used system for therapeutic gene delivery in the central nervous system (CNS) …