RNAi-BASED TARGETING COMPOUNDS AND USES THEREOF TO PREVENT ACQUIRED HEARING LOSS
AssigneeMUSC Foundation for Research Development
InventorSuhua SHA
Life of this patent
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Abstract
Described herein are compositions capable of targeting CaMKKβ and/or AMPK alpha and formulations thereof. Also described herein are methods of using the compositions and formulations thereof. In some embodiments, the compositions capable of targeting CaMKKβ and/or AMPK alpha and formulations thereof can prevent or treat outer hair cell loss, such as that which can occur as a result of auditory or chemical insult. In some embodiments, the compositions capable of targeting CaMKKβ and/or AMPK alpha and formulations thereof can treat or prevent acquired hearing loss.
Claims51
A method of treating or preventing an acquired (i) hearing loss, (ii) ototoxicity, (iii) outer hair cell damage or death, (iv) loss of an outer hair cell function, or a combination thereof comprising:
- a. administering an amount of an AMPK alpha targeting RNAi molecule to the subject in need thereof,
- b. administering an amount of a CaMKKβ targeting RNAi molecule to the subject in need thereof, or
- c. a combination thereof.
A method of reducing an amount of an AMPK alpha gene product, a CaMKKβ gene product, or both in a subject in need thereof, the method comprising:
- a. administering an amount of an AMPK alpha targeting RNAi molecule to the subject in need thereof,
- b. administering an amount of a CaMKKβ targeting RNAi molecule to the subject in need thereof, or
- c. a combination thereof.
A pharmaceutical formulation capable of treating or preventing an acquired (i) hearing loss, (ii) ototoxicity, (iii) outer hair cell damage or death, (iv) loss of an outer hair cell function, or a combination thereof comprising:
- a therapeutically effective amount of
- a. an AMPK alpha targeting RNAi molecule;
- b. a CaMKKβ targeting RNAi molecule;
- or
- c. both;
- and
- a pharmaceutically acceptable carrier.
A kit comprising:
- an AMPK alpha targeting RNAi molecule or a pharmaceutical formulation thereof, a CaMKKβ targeting RNAi molecule or a pharmaceutical formulation thereof;
- or both;
- and
- instructions fixed in a tangible medium of expression, wherein the instructions provide direction to administer the AMPK alpha targeting RNAi molecule or a pharmaceutical formulation thereof, the CaMKKβ targeting RNAi molecule or a pharmaceutical formulation thereof, or both to a subject in need thereof to
- a. treat or prevent an acquired
- i. hearing loss;
- ii. ototoxicity;
- iii. outer hair cell damage or death;
- iv loss of an outer hair cell;
- or
- v. a combination thereof;
- b. decrease or eliminate
- i. the amount of an AMPK alpha gene product;
- ii. the amount of an CaMKKβ gene product;
- iii gene expression, protein expression, or both of AMPK alpha isoform 1, AMPK alpha isoform 2, or both;
- iv. gene expression, protein expression, or both of CaMKKβ;
- or
- v. a combination thereof;
- or
- c. both
- in the subject.
Dependencies are inferred from the claim text.