Peripheral nerve injury in humans often leads to incomplete functional recovery. In this
review we discuss the potential for gene therapy to be used as a strategy alongside surgical …

We have previously reported that the removal of a 20-nucleotide sequence, termed the D
sequence, from both ends of the inverted terminal repeats (ITRs) in the adeno-associated …

The commonest severe inherited bleeding disorder in all ethnic groups worldwide is
hemophilia A, followed by hemophilia B. These are X-linked recessive disorders that result …

As our understanding of the genetic basis for inherited retinal disease has expanded, gene
therapy has advanced into clinical development. When the gene mutations associated with …

Duchenne muscular dystrophy (DMD) is a X-linked, progressive childhood myopathy
caused by mutations in the dystrophin gene, one of the largest genes in the genome. It is …

A drawback of gene therapy using adeno-associated virus (AAV) is the DNA packaging
restriction of the viral capsid (< 4.7 kb). Recent observations demonstrate oversized AAV …

Adeno‐associated viruses (AAVs) are the vector of choice for delivering gene therapies that
can cure inherited and acquired diseases. Clinical research on various AAV serotypes …

Adeno-associated viruses (AAVs) are preferred vectors for gene replacement therapy, as
they are non-pathogenic, non-inflammatory, induce stable transgene expression in …

Despite significant advances in novel treatments and approaches, cardiovascular disease
remains the leading cause of death globally. Gene therapy is a promising option for many …

Background: Gene therapy represents the therapeutic delivery of nucleic acid polymers into
patient cells with the aim of treating an underlying disease. Over the past 2 decades this new …