Monoclonal antibodies recognising
Adeno-associated virus (AAV)
Adeno-associated viruses A(AAV), a member
of the parvovirus family, are small single-stranded DNA viruses that infect
humans and some non-human primate species. Currently 13 human and non-human
primate AAV serotypes and 150 genotypes have been reported. AAV are currently
considered to be non-pathogenic and most people infected with AAV only develop
a mild immune response. Adeno-associated virus capsids carry genes into both
dividing and quiescent cells where they can insert genetic information into the
The different AAV serotypes have been shown
to demonstrate tropism
for specific tissues and cell types. This is due to the variations in their
amino acids sequences and capsid structure, which enables them to interact with
different host cell receptors. These characteristics of AAV have been exploited
for gene therapy and recombinant AAV virus have been engineered to carry
therapeutic genes into a diverse range of host cells.
Various clinical trials are currently ongoing
which utilise different AAV serotypes including: -
has been reported to show enhanced tropism for skeletal muscle, heart muscle,
retinal pigment epithelium and the central nervous system (CNS). AAV1 is
currently used in clinical trial studies for alpha-1 antitrypsin deficiency,
limb girdle muscular dystrophy, Duchenne muscular dystrophy and heart failure
demonstrates tropism for kidney tissue, retinal pigment
epithelium, photoreceptors and the CNS. AAV2 is currently used in clinical
trials studies for a wide range of conditions including hemophilia B, Duchenne
muscular dystrophy, Parkinson’s disease, age-related macular degeneration,
choroideremia and rheumatoid arthritis.
demonstrates tropism for retinal pigment epithelium, photoreceptors, lung
tissue, and the CNS. AAV5 is currently used in clinical trial studies for
haemophilia B and rheumatoid arthritis gene therapy.
demonstrates tropism for skeletal muscle and lung tissue. AAV6 has been used in
clinical trials for the treatment of severe heart failure.
demonstrates tropism for a wide range of tissues including liver, pancreas,
skeletal muscle, heart and CNS. AAV8 is currently used in clinical trials for a
range of conditions including hemophila A, hemophilia B, X-linked myotubular
myopathy and hepatitis C gene therapy.
demonstrates tropism for skeletal muscle, heart, liver, lung and the central
nervous system. AAV9 is currently used in clinical trial studies for Pompe
disease, Batten disease and spinal muscular atrophy gene therapy.