- Document Number:
20190336332
- Appl. No:
16/310084
- Application Filed:
June 15, 2017
- نبذة مختصرة :
A method of treating spinal muscular atrophy by inducing a heat shock response in a subject in need thereof is described. The heat shock response can be induced by heating the temperature of a tissue region of the subject above 37° C., or by administering a therapeutically effective amount of a heat shock inducing agent.
- Claim:
1. A method of treating spinal muscular atrophy (SMA) comprising inducing a heat shock response in a subject in need thereof.
- Claim:
2. The method of claim 1, wherein the heat shock response is induced by heating the temperature of a tissue region of the subject above 37° C.
- Claim:
3. The method of claim 1, wherein the heat shock response is induced by administering a therapeutically effective amount of a heat shock inducing agent.
- Claim:
4. The method of claim 3, wherein administration of the heat shock inducing agent increases full length SMN2 mRNA and full length SMN2 protein in neural cells of the subject by at least about 50%.
- Claim:
5. The method of claim 3, wherein administration of the heat shock inducing agent increases full length SMN2 mRNA and full length SMN2 protein in muscle cells of the subject by at least about 50%.
- Claim:
6. The method of claim 3, wherein administration of the heat shock inducing agent increases full length SMN2 mRNA and full length SMN2 protein in glial cells of the subject by at least about 50%.
- Claim:
7. The method of claim 3, wherein the heat shock inducing agent is a protein synthesis inhibitor.
- Claim:
8. The method of claim 3, wherein the heat shock inducing agent is a proteasome inhibitor.
- Claim:
9. The method of claim 3, wherein the heat shock inducing agent is serine protease inhibitor.
- Claim:
10. The method of claim 3, wherein the heat shock inducing agent is an Hsp90 inhibitor.
- Claim:
11. The method of claim 3, wherein the heat shock inducing agent is a non-steroidal anti-inflammatory drug.
- Claim:
12. The method of claim 3, wherein the heat shock inducing agent is a hydroxylamine derivative.
- Claim:
13. The method of claim 3, wherein the heat shock inducing agent is a co-inducer.
- Claim:
14. The method of claim 13, wherein the co-inducer is arimoclomol.
- Claim:
15. The method of claim 3, wherein the heat shock inducing agent is administered into the central nervous system.
- Claim:
16. The method of claim 3, wherein the heat shock inducing agent is administered into the cerebrospinal fluid.
- Claim:
17. The method of claim 3, wherein the head shock inducing agent is administered into muscle.
- Claim:
18. The method of claim 3, wherein the heat shock inducing agent is administered in drinking water.
- Claim:
19. The method of claim 1, wherein the subject is a human.
- Claim:
20. The method of claim 1, wherein the subject is a fetus and the heat shock response is induced in utero.
- Claim:
21. The method of claim 1, wherein the subject has been diagnosed with type I SMA.
- Claim:
22. The method of claim 1, wherein the subject has been diagnosed with type II SMA.
- Claim:
23. The method of claim 1, wherein the subject has been diagnosed with type III SMA.
- Claim:
24. The method of claim 1, wherein the subject has been diagnosed with type IV SMA.
- Current International Class:
61; 61; 61
- الرقم المعرف:
edspap.20190336332
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