Glutamic acid decarboxylase (gad) based delivery systems

A - Human Necessities – 61 – K

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Details

A61K 48/00 (2006.01) A61K 38/51 (2006.01) C12N 15/864 (2006.01)

Patent

CA 2409674

The invention provides methods and compositions for localized delivery of a vector comprising a therapeutic agent to a specific region of the brain that is overstimulated in neurodegenerative diseases. In particular, the invention provides methods and compositions used to deliver an adeno-associated virus vector (AAV) comprising a nucleotide sequence encoding glutamic acid decarboxylase (GAD) to cells in the subthalmic nucleus of the basal ganglia, mesaphilia and thalmus.

L'invention concerne des méthodes et compositions pour une administration localisée d'un vecteur comprenant un agent thérapeutique dans une région spécifique du cerveau qui est surexcitée en cas de maladie neurodégénérative. Cette invention concerne en particulier des méthodes et compositions pour administrer un vecteur viral adéno-associé (AAV), comprenant une séquence nucléotidique codant l'acide glutamique décarboxylase (GAD) aux cellules du noyau sous-thalamique des noyaux gris centraux, du mésaphilia et du thalamus.

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