Defective genes account for∼ 80% of the total of more than 7,000 diseases known to date.
Gene therapy brings the promise of a one-time treatment option that will fix the errors in …

Recombinant adeno-associated virus (rAAV) gene therapy has the potential to transform the
lives of patients with certain genetic disorders by increasing or restoring function to affected …

Bioengineering of viral vectors for therapeutic gene delivery is a pivotal strategy to reduce
doses, facilitate manufacturing, and improve efficacy and patient safety. Here, we …

Recombinant adeno-associated viruses (AAV) are under intensive investigation in
numerous clinical trials after they have emerged as a highly promising vector for human …

Adeno-associated virus (AAV) forms the basis for several commercial gene therapy products
and for countless gene transfer vectors derived from natural or synthetic viral isolates that …

While clinical gene therapy celebrates its first successes, with several products already
approved for clinical use and several hundreds in the final stages of the clinical approval …

Sarcopenia is a comprehensive degenerative disease with the progressive loss of skeletal
muscle mass with age, accompanied by the loss of muscle strength and muscle dysfunction …

Metabolic diseases are caused by a complex interplay between genetics and the
environment, yet many basic studies guiding our understanding of metabolism are confined …

Adeno-associated virus (AAV) vectors have gained tremendous attention as in vivo delivery
systems in gene therapy for inherited monogenetic diseases. First market approvals …

Skeletal muscle has gained recognition as an endocrine organ releasing myokines upon
contraction during physical exercise. These myokines exert both local and pleiotropic health …