Increasing Atoh1 life to drive sensorineural hair cell differentiation
AssigneeMassachusetts Eye and Ear Infirmary
InventorsAlbert Edge, Yen-Fu Cheng, Judith Kempfle, Dunia Abdul-Aziz
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Abstract
The present disclosure provides compositions and methods for treating subjects at risk for or with sensorineural hearing loss by modulating the rate of Atoh1 protein degradation to increase levels of Atoh1 protein.
Claims13
An expression vector comprising a polynucleotide encoding a human Atoh1 variant polypeptide, wherein the human Atoh1 variant polypeptide is a long-lived human Atoh1 variant polypeptide comprising a mutation at the amino acid position corresponding to amino acid 334 of SEQ ID NO:
- 1, and wherein the long-lived human Atoh1 variant polypeptide is at least 95% identical to SEQ ID NO:
- 1.
An isolated nucleic acid molecule comprising a nucleotide sequence encoding a human Atoh1 variant polypeptide, wherein the human Atoh1 variant polypeptide is a long-lived human Atoh1 variant polypeptide comprising a mutation at the amino acid position corresponding to amino acid position 334 of SEQ ID NO:
- 1, and wherein the long-lived human Atoh1 variant polypeptide is at least 95% identical to SEQ ID NO:
- 1.
Dependencies are inferred from the claim text.