Novel gene therapy construct for cln2 disease
AssigneeThe Children's Hospital of Philadelphia
InventorsBeverly DAVIDSON, Paul RANUM, Yonghong CHEN, Megan S. KEISER, Luis TECEDOR
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Abstract
Provided herein are methods of expressing a tripeptidyl peptidase 1 (TPP1) in a neuron. Also provided herein is a modified adeno-associated virus (AAV) encoding a TPP1 under the control of a promoter operable in a neuron or ependyma cell, and not in dorsal root ganglia.
Claims45
A method of expressing a tripeptidyl peptidase 1 (TPP1) in a neuron and/or ependyma cells of a subject, comprising administering to the subject a modified adeno-associated virus (AAV) encoding a TPP1 under the control of a promoter operable in said neuron, such as central nervous system neurons, or said ependymal cell, wherein said AAV comprises a modified capsid protein comprising the targeting sequence ERDRTRG (SEQ ID NO:
- 3), and wherein said modified AAV does not target dorsal root ganglia.
A modified adeno-associated virus (AAV) encoding a TPP1 under the control of a promoter operable in a neuron or ependyma cell, wherein said AAV comprises a modified capsid protein comprising the targeting sequence ERDRTRG (SEQ ID NO:
- 3), and wherein said modified AAV does not target dorsal root ganglia.
Use of a modified adeno-associated virus (AAV) encoding a tripeptidyl peptidase 1 (TPP1) under the control of a promoter operable in a neuron, wherein said AAV comprises a modified capsid protein comprising the targeting sequence ERDRTRG (SEQ ID NO:
- 3), for expressing TPP1 in a neuron or ependymal cell, wherein said modified AAV does not target dorsal root ganglia.
Use of a modified adeno-associated virus (AAV) encoding a tripeptidyl peptidase 1 (TPP1) under the control of a promoter operable in a neuron or ependymal cell, wherein said AAV comprises a modified capsid protein comprising the targeting sequence ERDRTRG (SEQ ID NO:
- 3), for treating late infantile neuronal ceroid lipofusincosis type 2 (CLN2) disease/Batten disease in a subject afflicted therewith, wherein said modified AAV does not target dorsal root ganglia.
Use of a modified adeno-associated virus (AAV) encoding a tripeptidyl peptidase 1 (TPP1) under the control of a promoter operable in a neuron or ependymal cell, wherein said AAV comprises a modified capsid protein comprising the targeting sequence ERDRTRG (SEQ ID NO:
- 3), in the preparation of a medicament for treating late infantile neuronal ceroid lipofusincosis type 2 (CLN2) disease/Batten disease in a subject afflicted therewith, wherein said modified AAV does not target dorsal root ganglia.
Dependencies are inferred from the claim text.