"adeno-associated virus" is a small single strand DNA virus. It belongs to the family of the Parvoviridae and in the genus Dependovirus. The viral particle is composed of one positive or negative polarity strand of DNA in a capsid. The mean size of an AAV particle is 18 to 22 nm. AAVs are the only non-autonomous parvoviruses. When we speak of "rAAV", this means the recombinant AAV virus, i.e. modified to become a vector (and thus is no longer infectious).

both genes located at a precise spot on a chromosome, one inherited from the father and the other from the mother they are located at exactly the same place.

a different origin from the original organism.

graft of cells, tissues or organs from a donor. Immunological compatibility between donor and recipient is not guaranteed. There are frequent risks of grafted organ rejection by the recipient organism.

muscular atrophy, reduction of muscle volume.

referring to analysis methods.

animal with a spontaneous or experimentally induced disease, used to study the human disease or for pharmacolgical studies.

substances that decrease or suppress pain.

antibodies targeted specifically to the muscle receptor to acetylcholine. These antibodies bind to the receptors preventing the attachment of acetylcholine and the propagation of nervous conduction.

protein produced by the immune system (especially B lymphocytes) in response to the penetration of a foreign molecule in the organism. It is tightly bound by the antibody to inactivate it or to label it for elimination by the organism.

substance that blocks the action of acetylcholinesterase, preventing the degradation of acetylcholine.

molecule inducing production of antibodies specifically targeted against it.

RNA complementary to messenger RNA. When combined with messenger RNA, translation of the latter is blocked.

cells providing support functions to the central nervous system.

Adenosine triphosphate

volume reduction of an organ or tissue due to varied pathological lesions.

antibody produced against one’s self (and not against a foreign substance).

disease involving the production of antibodies against a part of one’s self (auto-antibodies).

graft of cells, tissues or organs coming from one's own body.

mode of transmission of a hereditary disease in which the patient received a chromosome from a sick parent bearing the gene anomaly. The involvement of only one parent by the disease is sufficient to transmit the disease to the child with a risk of 1/2 (50%) at each pregnancy.

mode of transmission of a hereditary disease in which the patient received two chromosomes bearing the gene anomaly, one from each parent. For these parents, the risk of transmitting an autosomal recessive disease is 1/4 (25%) at each pregnancy.

AAV is incapable of carrying out a replication cycle in the course of cell infection. The presence of another virus, called a helper (auxiliary), provides additional functions needed for viral replication and leads to a productive infectious cycle. This dependency is the origin of the genus name of AAVs (dependovirus).