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AssigneeResearch Institute At Nationwide Children's Hospital
InventorZarife Sahenk
Estimating the term…
Provided are gene therapy vectors, such as adeno-associated virus (AAV), compositions and methods for treatment of diseases caused by misfolded proteins. The disclosed rAAV comprise a nucleotide sequence encoding the BCL2 Associated Athanogene 3 (BAG-3) protein, and methods of administering these rAAV to treat of diseases and disorders associated with protein misfolding and/or aggregation in o a subject in need, which results in increased targeting of aggregation prone proteins for degradation through the BAG3-mediated selective macroautophagy pathway, thereby restoring proteostasis. The disclosed gene therapy vectors, such as rAAV constructs also are used for treatment of inclusion body myositis (IBM) associated with Paget disease of bone and frontotemporal dementia (IBMPFD) and multisystem proteinopathy.
A polynucleotide sequence comprising a transcriptional control element;
A polynucleotide sequence comprising a polynucleotide sequence having or 90% identity to nucleotides 1 to 3245 of SEQ ID NO:
A polynucleotide sequence comprising a polynucleotide sequence comprising nucleotides 1 to 3245 of SEQ ID NO:
The method, use or composition of any one of claims 33-35, wherein the neurodegenerative disease is ALS, and wherein the misfolded protein or protein aggregates associated with the ALS is SOD1.
The method, use or composition of any one of claims 22-241, further comprising administering a second nucleic acid encoding a NT-3 polypeptide;
Dependencies are inferred from the claim text.