US Patent 11219696 Delivery of polynucleotides using recombinant AAV9
Patent 11219696 was granted and assigned to Nationwide Children's Hospital on January, 2022 by the United States Patent and Trademark Office.
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The present invention relates to Adeno-associated virus 9 methods and materials useful for systemically delivering polynucleotides across the blood brain barrier. Accordingly, the present invention also relates to methods and materials useful for systemically delivering polynucleotides to the central and peripheral nervous systems. The present invention also 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 spinal muscle atrophy and amyotrophic lateral sclerosis as well as Pompe disease and lysosomal storage disorders. Use of the methods and materials is also indicated, for example, for treatment of Rett syndrome.
US Patent 11219696 Delivery of polynucleotides using recombinant AAV9
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