Intrathecal Delivery of Recombinant Adeno-Associated Virus 9

Abstract: the present invention relates to adeno-associated virus type 9 methods and materials useful for intrathecal delivery of polynucleotides. use of the methods and materials is indicated. for example. for treatment of lower motor neuron diseases such as sma and als as well as pompe disease and lysosomal storage disorders.

Inventor(s): Brian K. Kaspar, Arthur Burghes, Paul Porensky

CPC Classification: A61K48/0075 (Medicinal preparations containing genetic material which is inserted into cells of the living body to treat genetic diseases; Gene therapy)

Search for rejections for patent application number 20250205368