Brain delivery is one of the major challenges in drug development because of the high
number of patients suffering from neural diseases and the low efficiency of the treatments …

Gene therapy is at the cusp of a revolution for treating a large spectrum of CNS disorders by
providing a durable therapeutic protein via a single administration. Adeno-associated virus …

In recent years, vast amounts of data on the mechanisms of neural de-and regeneration
have accumulated. However, only in disproportionally few cases has this led to efficient …

Lysosomal storage diseases (LSDs) comprise a diverse group of monogenetic disorders
with complex clinical phenotypes that include both systemic and central nervous system …

Various regions of the brain have been successfully transduced by recombinant adeno‐
associated virus (rAAV) vectors with no detected toxicity. When using the cytomegalovirus …

Protein transduction with cell penetrating peptides over the past several years has been
shown to be an effective way of delivering proteins in vitro and now several reports have …

Rare monogenic disorders such as lysosomal diseases have been at the forefront in the
development of novel treatments where therapeutic options are either limited or unavailable …

Plasmid DNA and adenovirus vectors currently used in cardiovascular gene therapy trials
are limited by low efficiency and short-lived transgene expression, respectively …

Recombinant adeno-associated virus (rAAV) is derived from a small human parvovirus with
an excellent safety profile. In addition, this viral vector efficiently transduces and supports …

Adeno-associated virus (AAV) vectors are capable of delivering a therapeutic gene to the
mouse brain that can result in long-term and widespread protein production. However, the …