FIELD: medicine, pharmaceutics.
SUBSTANCE: claimed group of inventions relates to field of medicine. Claimed is method of relieving at least one symptom of spinal muscular atrophy in subject, which includes introduction to subject of antisense compound, containing antisense oligonucleotide, complementary to intron 7 of pre-mRNA, coding human SMN2. Claimed is application of such antisense compound in production of medication for relieving or treatment of at least one symptom of spinal muscular atrophy in subject.
EFFECT: claimed group of inventions provides effective means and methods for relieving spinal muscular atrophy in subject.
13 cl, 13 dwg, 14 tbl, 14 ex
| Title | Year | Author | Number |
|---|---|---|---|
| COMPOSITIONS AND METHODS FOR MODULATION OF SMN2 SPLICING IN A SUBJECT | 2019 |
|
RU2793459C2 |
| COMPOSITIONS AND METHODS OF SML2 SPLICING MODULATION IN SUBJECT | 2010 |
|
RU2683772C2 |
| COMPOSITIONS FOR MODULATING EXPRESSION OF SOD-1 | 2015 |
|
RU2704619C2 |
| COMPOSITIONS AND METHODS FOR MODULATING PKK EXPRESSION | 2015 |
|
RU2703411C2 |
| COMPOSITIONS AND METHODS OF MODULATING APOLIPOPROTEIN C-III EXPRESSION | 2014 |
|
RU2650510C2 |
| COMPOSITIONS AND METHODS FOR MODULATING EXPRESSION OF APOLIPOPROTEIN (A) | 2014 |
|
RU2824214C1 |
| COMPOSITIONS AND METHODS FOR MODULATING COMPLEMENT FACTOR EXPRESSION B | 2015 |
|
RU2701645C2 |
| MODULATION OF HUNTINGTIN EXPRESSION | 2010 |
|
RU2562861C2 |
| COMPOUNDS AND METHODS FOR MODULATING EXPRESSION OF DYSTROPHIC MIOTONIN-PROTEINKINASE (DMPK) | 2014 |
|
RU2690333C2 |
| COMPOSITIONS AND METHODS FOR MODULATING HBV AND TTR EXPRESSION | 2014 |
|
RU2670614C9 |
