AR097889A1 - COMPOSITIONS AND METHODS TO INHIBIT THE EXPRESSION OF GEN LECT2 - Google Patents

COMPOSITIONS AND METHODS TO INHIBIT THE EXPRESSION OF GEN LECT2

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Publication number
AR097889A1
AR097889A1 ARP140103664A ARP140103664A AR097889A1 AR 097889 A1 AR097889 A1 AR 097889A1 AR P140103664 A ARP140103664 A AR P140103664A AR P140103664 A ARP140103664 A AR P140103664A AR 097889 A1 AR097889 A1 AR 097889A1
Authority
AR
Argentina
Prior art keywords
chain
lect2
nucleotides
seq
expression
Prior art date
Application number
ARP140103664A
Other languages
Spanish (es)
Inventor
Bettencourt Brian
Fitzgerald Kevin
Hinkle Gregory
Sehgal Alfica
Original Assignee
Alnylam Pharmaceuticals Inc
Priority date (The priority date is an assumption and is not a legal conclusion. Google has not performed a legal analysis and makes no representation as to the accuracy of the date listed.)
Filing date
Publication date
Application filed by Alnylam Pharmaceuticals Inc filed Critical Alnylam Pharmaceuticals Inc
Publication of AR097889A1 publication Critical patent/AR097889A1/en

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Abstract

Reivindicación 1: Un ácido ribonucleico de cadena doble (ARNcd) para inhibir la expresión de LECT2, caracterizado porque dicho ARNcd comprende una cadena orientada en el sentido del marco de lectura que es de 15 - 30 pares de bases de longitud y una cadena antisentido que es de 15 - 30 pares de bases de longitud y la cadena antisentido es complementaria de por lo menos 15 nucleótidos contiguos de la SEQ ID Nº 1. Reivindicación 4: Un ARNi de cadena doble (ARNcd) que comprende una cadena orientada en el sentido del marco de lectura complementaria de una cadena antisentido, caracterizado porque dicha cadena antisentido comprende una región de complementariedad con un transcripto de ARN de LECT2 que comprende la SEQ ID Nº 1 o una secuencia de nucleótidos que tiene una sustitución de A por G en la posición de nucleótido 373 de la SEQ ID Nº 1, en donde cada cadena tiene entre aproximadamente 14 y aproximadamente 30 nucleótidos, en donde dicho ARNcd está representada por la fórmula: cadena orientada en sentido del marco de lectura: 5 nₚ-Nₐ-(XXX)ⁱ-Nᵇ-YYY-Nᵇ-(ZZZ)ʲ-Nₐ-nq 3 cadena antisentido: 3 nₚ-Nₐ-(XXX)ₖ-Nᵇ-YYY-Nᵇ-(ZZZ)ₗ-Nₐ-nq 5 en donde: i, j, k y I son cada uno de manera independiente 0 ó 1; p, p, q y q son cada uno de manera independiente 0 - 6; cada Nₐ y Nₐ representa de manera independiente una secuencia de oligonucleótidos que comprende 0 - 25 nucleótidos que están modificados o no modificados, o combinaciones de los mismos, donde cada secuencia comprende por lo menos dos nucleótidos modificados de manera diferente; cada Nᵇ y Nᵇ representa de manera independiente una secuencia de oligonucleótidos que comprende 0 - 10 nucleótidos que están modificados o no modificados, o combinaciones de los mismos; cada nₚ, nₚ, nq y nq representa de manera independiente un nucleótido saliente; XXX, YYY, ZZZ, XXX, YYY, y ZZZ representan cada uno de manera independiente un motivo de tres modificaciones idénticas en tres nucleótidos consecutivos; las modificaciones en Nᵇ difieren de la modificación en Y y las modificaciones en Nᵇ difieren de la modificación en Y. Reivindicación 35: Una composición farmacéutica para inhibir la expresión de un gen LECT2, caracterizada porque dicha composición comprende el ARNcd de cualquiera de las reivindicaciones 1 a 33. Reivindicación 49: Un método para inhibir la expresión de LECT2 en una célula, caracterizado porque dicho método comprende: (a) introducir en la célula el ARNcd de cualquiera de las reivindicaciones 1 - 33, y (b) mantener la célula producida en el paso (a) por un tiempo suficiente como para obtener la degradación del transcripto de ARNm de un gen LECT2, inhibiendo de esa manera la expresión del gen LECT2 en la célula. Reivindicación 58: Un método de tratamiento de una amiloidosis LECT2, caracterizado porque comprende administrar a un sujeto que necesita de dicho tratamiento un ácido ribonucleico de cadena doble (ARNcd), en donde dicho ARNcd comprende una cadena orientada en el sentido del marco de lectura que es de 15 - 30 pares de bases de longitud y una cadena antisentido que es de 15 - 30 pares de bases de longitud y la cadena antisentido es complementaria de por lo menos 15 nucleótidos contiguos de la SEQ ID Nº 1 o una secuencia de nucleótidos que tiene una sustitución de A por G en la posición de nucleótido 373 de la SEQ ID Nº 1.Claim 1: A double-chain ribonucleic acid (dsRNA) for inhibiting the expression of LECT2, characterized in that said dsRNA comprises a chain oriented in the direction of the reading frame that is 15-30 base pairs in length and an antisense chain that It is 15-30 base pairs in length and the antisense chain is complementary to at least 15 contiguous nucleotides of SEQ ID No. 1. Claim 4: A double-stranded RNAi (dsRNA) comprising a chain oriented in the direction of complementary reading frame of an antisense chain, characterized in that said antisense chain comprises a region of complementarity with an RNA transcript of LECT2 comprising SEQ ID No. 1 or a nucleotide sequence having a substitution of A for G at the position of nucleotide 373 of SEQ ID No. 1, where each chain has between about 14 and about 30 nucleotides, wherein said cRNA is represented by the fo formula: chain-oriented chain reading: 5 nₚ-Nₐ- (XXX) ⁱ-Nᵇ-YYY-Nᵇ- (ZZZ) ʲ-Nₐ-nq 3 antisense chain: 3 nₚ-Nₐ- (XXX) ₖ-Nᵇ -YYY-Nᵇ- (ZZZ) ₗ-Nₐ-nq 5 where: i, j, k and I are each independently 0 or 1; p, p, q and q are each independently 0-6; each Nₐ and Nₐ independently represents an oligonucleotide sequence comprising 0-25 nucleotides that are modified or unmodified, or combinations thereof, where each sequence comprises at least two differently modified nucleotides; each Nᵇ and Nᵇ independently represents an oligonucleotide sequence comprising 0-10 nucleotides that are modified or unmodified, or combinations thereof; each nₚ, nₚ, nq and nq independently represents an outgoing nucleotide; XXX, YYY, ZZZ, XXX, YYY, and ZZZ each independently represent a reason for three identical modifications in three consecutive nucleotides; the modifications in Nᵇ differ from the modification in Y and the modifications in Nᵇ differ from the modification in Y. Claim 35: A pharmaceutical composition for inhibiting the expression of a LECT2 gene, characterized in that said composition comprises the cDNA of any of claims 1 a 33. Claim 49: A method for inhibiting the expression of LECT2 in a cell, characterized in that said method comprises: (a) introducing into the cell the cRNA of any of claims 1-33, and (b) maintaining the cell produced in step (a) for sufficient time to obtain degradation of the mRNA transcript of an LECT2 gene, thereby inhibiting the expression of the LECT2 gene in the cell. Claim 58: A method of treating a LECT2 amyloidosis, characterized in that it comprises administering to a subject in need of said treatment a double-stranded ribonucleic acid (dsRNA), wherein said dsRNA comprises a chain oriented in the direction of the reading frame that it is 15-30 base pairs in length and an antisense chain that is 15-30 base pairs in length and the antisense chain is complementary to at least 15 contiguous nucleotides of SEQ ID No. 1 or a nucleotide sequence that It has a substitution of A for G at nucleotide position 373 of SEQ ID No. 1.

ARP140103664A 2013-10-02 2014-10-02 COMPOSITIONS AND METHODS TO INHIBIT THE EXPRESSION OF GEN LECT2 AR097889A1 (en)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201361885693P 2013-10-02 2013-10-02

Publications (1)

Publication Number Publication Date
AR097889A1 true AR097889A1 (en) 2016-04-20

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Application Number Title Priority Date Filing Date
ARP140103664A AR097889A1 (en) 2013-10-02 2014-10-02 COMPOSITIONS AND METHODS TO INHIBIT THE EXPRESSION OF GEN LECT2

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AR (1) AR097889A1 (en)

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN110646615A (en) * 2019-08-27 2020-01-03 南方医科大学 Biological marker and treatment target of hepatic fibrosis and application thereof

Cited By (1)

* Cited by examiner, † Cited by third party
Publication number Priority date Publication date Assignee Title
CN110646615A (en) * 2019-08-27 2020-01-03 南方医科大学 Biological marker and treatment target of hepatic fibrosis and application thereof

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