Materials and methods for neurofibromin 2/merlin (nf2) gene therapy
AssigneeResearch Institute At Nationwide Children's Hospital
InventorsLong-Sheng Chang, Kevin Flanigan, Shibi Likhite, Kathrin Meyer
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Abstract
The present disclosure relates to methods of treating conditions associated with a need for Merlin protein, for example due to a defective Neurofibromin 2/Merlin (NF2) gene as in neurofibromatosis type 2 (NF2). In particular. the disclosure provides gene therapy vectors to specifically treat loss of expression of the Merlin protein or reduced Merlin protein levels.
Claims22
A method of delivering a transgene encoding a Merlin protein with tumor suppressor activity to a subject in need of Merlin protein tumor suppressor activity, wherein the method comprises administering a gene therapy vector comprising the transgene to the subject by intravenous delivery, intratumoral delivery or intrathecal delivery.
A transgene comprising the polynucleotide of SEQ ID NO:
- 1, 6, 7, 8, or 9, or a polynucleotide at least 90%, 91%, 92%, 93%, 94%, 95%, 96%, 97%, 98%, or 99% identical to SEQ ID NO:
- 1, 6, 7, 8 or 9 that encodes a Merlin protein with tumor suppressor activity.
A transgene comprising the polynucleotide of SEQ ID NO:
- 16, 17, 18, 19, 20, or a polynucleotide at least 90%, 91%, 92%, 93%, 94%, 95%, 96%, 97%, 98%, or 99% identical to SEQ ID NO:
- 16, 17, 18, 19, 20, that encodes a phosphorylation-resistant Merlin protein with tumor suppressor activity.
A recombinant adeno-associated virus (rAAV) with a genome comprising a transgene comprising the polynucleotide of SEQ ID NO:
- 1, 6, 7, 8, or 9, or a polynucleotide at least 90%, 91%, 92%, 93%, 94%, 95%, 96%, 97%, 98%, or 99% identical to SEQ ID NO:
- 1, 6, 7, 8 or 9 that encodes a Merlin protein with tumor suppressor activity.
A recombinant adeno-associate virus (rAAV) with a genome comprising a transgene comprises the polynucleotide of SEQ ID NO:
- 16, 17, 18, 19, 20, or a polynucleotide at least 90%, 91%, 92%, 93%, 94%, 95%, 96%, 97%, 98%, or 99% identical to SEQ ID NO:
- 16, 17, 18, 19, 20, that encodes a phosphorylation-resistant Merlin protein with tumor suppressor activity.
Dependencies are inferred from the claim text.