Duchenne muscular dystrophy (DMD) is a rare neuromuscular disease affecting 1: 5000
newborn males. No cure is currently available, but gene addition therapy, based on the …

Next-generation DNA vectors for cancer immunotherapies and vaccine development require
promoters eliciting predefined transcriptional activities specific to target cell types, such as …

Objective Pompe disease (PD) is caused by deficiency of the lysosomal enzyme acid α-
glucosidase (GAA), leading to progressive glycogen accumulation and severe myopathy …

Duchenne muscular dystrophy (DMD) is a severe neuromuscular disease caused by Dmd
mutations, resulting in the absence of dystrophin in skeletal muscle, and a greater …

Robust expression of shortened, functional dystrophin provided impetus to develop adeno-
associated virus (AAV)–based constructs for clinical application. Because several cassettes …

Duchenne muscular dystrophy (DMD) is an X-linked recessive disease that affects 1: 5,000
live male births and is characterized by muscle wasting. By the age of 13 years, affected …

Transduction of endothelial cells (EC) with a vector that expresses apolipoprotein AI (APOAI)
reduces atherosclerosis in arteries of fat-fed rabbits. However, the effects on atherosclerosis …

Via the peripheral and autonomic nervous systems, the spinal cord directly or indirectly
connects reciprocally with many body systems (muscular, intengumentary, respiratory …

The human musculature is a promising and pivotal target for human gene therapy, owing to
numerous diseases that affect this tissue and that are often monogenic, making them …

Viral vectors based gene therapy is often compromised by adverse immunological reactions
raising safety concerns. Hence, improved design and development of non-viral vectors with …