COMPOSITIONS AND METHODS FOR TREATMENT OF SPINAL MUSCULAR ATROPHY
Disclosed herein are compounds, compositions and methods for treatment of diseases and disorders, including spinal muscular atrophy.
1 .- 70 . (canceled)
71 . A method comprising administering at least one GF/IGF-1 axis modulator to a subject having spinal muscular atrophy (SMA).
72 . The method of claim 71 , wherein at least one GF/IGF-1 axis modulator is a GF/IGF-1 axis molecule.
73 . The method of claim 72 , wherein at least one GF/IGF-1 axis modulator increases the activity and/or amount of insulin-like growth factor 1 (IGF-1) in the subject.
74 . The method of claim 73 , wherein at least one GF/IGF-1 axis modulator is insulin-like growth factor 1 (IGF-1).
75 . The method of claim 71 , wherein at least one GF/IGF-1 axis modulator is IGF-binding-protein acid labile subunit (IGFALS).
76 . The method of claim 71 , wherein at least one GF/IGF-1 axis modulator is growth hormone (GH).
77 . The method of claim 71 , wherein at least one GF/IGF-1 axis modulator is insulin-like growth factor binding protein 3 (IGFBP3).
78 . The method of claim 71 , wherein at least one GF/IGF-1 axis modulator is a gene encoding a GF/IGF-1 axis molecule.
79 . The method of claim 71 , wherein at least one GF/IGF-1 axis modulator is a gene encoding IGF-binding-protein acid labile subunit (IGFALS).
80 . The method of claim 71 , wherein the at least one GF/IGF-1 axis modulator is administered systemically.
81 . The method of claim 71 , wherein at least one growth hormone axis modulator is administered by intraperitoneal injection.
82 . The method of claim 71 , wherein at least one growth hormone axis modulator is administered by subcutaneous injection.
83 . The method of claim 71 , wherein at least one growth hormone axis modulator is administered by intramuscular injection.
84 . The method of claim 71 , wherein at least one growth hormone axis modulator is administered into the cerebrospinal fluid.
85 . The method of claim 71 , comprising administering at least one antisense oligonucleotide to the subject having spinal muscular atrophy.
86 . The method of claim 85 , wherein the antisense compound comprises an antisense oligonucleotide complementary to a nucleic acid encoding human SMN2.
87 . The method of claim 86 , wherein the oligonucleotide is complementary to a portion of intron 7 of the nucleic acid encoding human SMN2.
88 . The method of claim 87 , wherein the antisense oligonucleotide is at least 90% complementary to the nucleic acid encoding human SMN2.
89 . The method of claim 88 , wherein the antisense oligonucleotide is fully complementary to the nucleic acid encoding human SMN2.
90 . The method of claim 89 , wherein the oligonucleotide has a nucleobase sequence comprising at least 10 contiguous nucleobases of the nucleobase sequence SEQ ID NO: 1.