Inhibitor of miR-129 and uses thereof
The present invention relates to an inhibitor of miR-129, relative compounds and pharmaceutical compositions for use in the treatment and/or prevention of amyotrophic lateral sclerosis and Alzheimer's disease. The invention also relates to a method for the diagnosis and/or prognosis of Alzheimer's disease in a subject or to identify a subject at risk to develop amyotrophic lateral sclerosis or Alzheimer's disease and to a method for the measuring the efficacy of a therapy for amyotrophic lateral sclerosis or for Alzheimer's disease and relative kits.
1 . A method for treating amyotrophic lateral sclerosis comprising administering a morpholino antisense oligonucleotide inhibitor of miR-129 to a subject diagnosed with amyotrophic lateral sclerosis, wherein said morpholino antisense oligonucleotide inhibitor of miR-129 comprises the sequence of SEQ ID NO: 3.
2 . The method according to claim 1 , wherein said morpholino antisense oligonucleotide inhibitor of miR-129 is:
a single-stranded or double-stranded nucleic acid molecule, an siRNA molecule, an antisense oligonucleotide, derivatives and mixtures thereof.
3 . The method of claim 1 , wherein the morpholino antisense oligonucleotide inhibitor of miR-129 is administered to the subject intracerebroventricularly, orally, rectally, transmucosal, intestinally, enterally, through inhalation, intraventricularly, intraperitoneally, intranasally, or parenterally.
4 . The method of claim 1 , wherein the subject has SOD1-associated ALS.
5 . The method of claim 1 , wherein said morpholino antisense oligonucleotide inhibitor of miR-129 consists the sequence of SEQ ID NO: 3.