ALNYLAM PHARMACEUTICALS, INC.;ICAHN SCHOOL OF MEDICINE AT MOUNT SINAI
发明人:
申请号：
ARP130101155
公开号：
AR090641A1
申请日:
2013.04.10
申请国别(地区):
AR
年份:
2014
代理人:
摘要：
Claim 1: a double stranded ribonucleic acid (dsRNA) to inhibit the expression of alas1, wherein Said dsRNA comprises a chain and a Chain of Sense antisense 15 - 30 base pairs in length and is complementary to the antisense Strand at least 15 contiguous nucleotides of SEQ I D no. 1 or 382.A Pharmaceutical composition claim 19: to inhibit the expression of a Gene alas1, where the composition comprises the dsRNA according to any one of claims 1 to 17.Claim 32: a method of inhibiting the expression of alas1 in a cell, comprising: (a) introducing the dsRNA in the cell according to claim 1 - 17 to 78 / 98 and (b) maintain the cell of Step (a) for a Time sufficient to obtain the Degr The adaci u00f3n mRNA Transcript of a Gene alas1, inhibiting the expression of the Gene in the cell alas1.Claim 42: a method of treating a disorder associated with the expression of alas1 that includes administering to a subject in need of such treatment a therapeutically effective amount of (i) The dsRNA according to any one of claims 1 - 1767 - 72 to 78 - 98 or (ii) the composition according to any of claims 1 - 31 / 99 - 111.Claim 151: a method of treating a subject with a high wing and / or PBG, which comprises the Administration to the subject of double stranded ribonucleic acid (d5arn)Where such dsRNA includes a Chain of Sense and antisense Chain of 15 - 30 base pairs in length and is complementary to the antisense Strand at least 15 contiguous nucleotides of SEQ ID no. 1 and SEQ ID no. 382.Reivindicación 1: Un ácido ribonucleico bicatenario (dsARN) para inhibir la expresión de ALAS1, en donde dicho dsARN comprende una cadena de sentido y una cadena antisentido 15 - 30 pares de bases de longitud y la cadena antisentido es complementaria a al menos 15 nucleótidos contiguos de SEQ ID Nº 1 ó 382. Reivindicación 19: Una composición farmacéutica para inhibir la expresión de un gen ALAS1, donde la composición comprende el dsARN de acuerdo con cualquiera de las reivindicaciones 1 - 17. Reivi
