CL2017002027A1 - Arni variante. - Google Patents

Arni variante.

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Publication number
CL2017002027A1
CL2017002027A1 CL2017002027A CL2017002027A CL2017002027A1 CL 2017002027 A1 CL2017002027 A1 CL 2017002027A1 CL 2017002027 A CL2017002027 A CL 2017002027A CL 2017002027 A CL2017002027 A CL 2017002027A CL 2017002027 A1 CL2017002027 A1 CL 2017002027A1
Authority
CL
Chile
Prior art keywords
sequence
arni
recombinating
memory
huntington
Prior art date
Application number
CL2017002027A
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English (en)
Inventor
Sergio Pablo Sardi
Catherine O'riordan
Lisa M Stanek
Adam Palermo
Brenda Richards
Antonius Song
Original Assignee
Genzyme Corp
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.)
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Publication date
Application filed by Genzyme Corp filed Critical Genzyme Corp
Publication of CL2017002027A1 publication Critical patent/CL2017002027A1/es

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    • C12N15/113—Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides; Antisense DNA or RNA; Triplex- forming oligonucleotides; Catalytic nucleic acids, e.g. ribozymes; Nucleic acids used in co-suppression or gene silencing
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    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P25/00—Drugs for disorders of the nervous system
    • A61P25/28—Drugs for disorders of the nervous system for treating neurodegenerative disorders of the central nervous system, e.g. nootropic agents, cognition enhancers, drugs for treating Alzheimer's disease or other forms of dementia
    • A—HUMAN NECESSITIES
    • A61—MEDICAL OR VETERINARY SCIENCE; HYGIENE
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    • A61P35/00—Antineoplastic agents
    • A—HUMAN NECESSITIES
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    • A61P—SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
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    • A61P9/10—Drugs for disorders of the cardiovascular system for treating ischaemic or atherosclerotic diseases, e.g. antianginal drugs, coronary vasodilators, drugs for myocardial infarction, retinopathy, cerebrovascula insufficiency, renal arteriosclerosis
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    • C12N2310/53—Physical structure partially self-complementary or closed
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    • C12N2710/00011—Details
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    • C12N2740/00—Reverse transcribing RNA viruses
    • C12N2740/00011—Details
    • C12N2740/10011—Retroviridae
    • C12N2740/15011—Lentivirus, not HIV, e.g. FIV, SIV
    • C12N2740/15041—Use of virus, viral particle or viral elements as a vector

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  • Health & Medical Sciences (AREA)
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  • Biomedical Technology (AREA)
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Abstract

<p>SE PROPORCIONAN EN LA PRESENTE MEMORIA MOLÉCULAS DE ARNi QUE INCLUYEN UNA PRIMERA CADENA QUE CONTIENE UNA SECUENCIA GUÍA Y UNA SEGUNDA CADENA QUE COMPRENDE UNA SECUENCIA NO GUÍA EN DONDE LA SECUENCIA NO GUÍA CONTIENE UNA PROTUBERANCIA OPUESTA A LA REGIÓN SEMILLA DE LAS SECUENCIAS GUÍA; P. EJ., OPUESTA A LA SECUENCIA DE ESCISIÓN. EN ALGUNOS ASPECTOS, LA INVENCIÓN PROPORCIONA ARNi PARA EL TRATAMIENTO DE LA ENFERMEDAD DE HUNTINGTON. ADICIONALMENTE SE PROPORCIONAN EN LA PRESENTE MEMORIA CASETES DE EXPRESIÓN, VECTORES (P, EJ., VECTORES DE rAAV, ADENOVIRALES RECOMBINANTES, LENTIVIRALES RECOMBINANTES, Y DE HSV RECOMBINANTES), CÉLULAS, PARTÍCULAS VIRALES, Y COMPOSICIONES FARMACÉUTICAS QUE CONTIENEN EL ARNi. TAMBIÉN SE PROPORCIONAN ADICIONA(MENTE EN LA PRESENTE MEMORIA MÉTODOS Y KITS RELACIONADOS CON EL USO DEL ARNi, POR EJEMPLO, PARA EL TRATAMIENTO DE LA ENFERMEDAD DE HUNTINGTON.</p>
CL2017002027A 2015-02-10 2017-08-08 Arni variante. CL2017002027A1 (es)

Applications Claiming Priority (1)

Application Number Priority Date Filing Date Title
US201562114578P 2015-02-10 2015-02-10

Publications (1)

Publication Number Publication Date
CL2017002027A1 true CL2017002027A1 (es) 2018-04-20

Family

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CL2017002027A CL2017002027A1 (es) 2015-02-10 2017-08-08 Arni variante.

Country Status (25)

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US (4) US10450563B2 (es)
EP (1) EP3256588A2 (es)
JP (2) JP2018506304A (es)
KR (1) KR102670852B1 (es)
CN (1) CN107438671B (es)
AR (1) AR103646A1 (es)
AU (3) AU2016219396B2 (es)
CA (2) CA3255657A1 (es)
CL (1) CL2017002027A1 (es)
CO (1) CO2017009083A2 (es)
CR (1) CR20170406A (es)
EA (1) EA201791805A1 (es)
EC (1) ECSP17059343A (es)
HK (1) HK1247641A1 (es)
IL (1) IL253893B (es)
MA (1) MA40819B1 (es)
MX (2) MX2017010369A (es)
MY (2) MY181458A (es)
PE (1) PE20171382A1 (es)
PH (1) PH12017501432A1 (es)
SG (3) SG10201907396XA (es)
TN (1) TN2017000354A1 (es)
TW (1) TWI781079B (es)
UY (1) UY36554A (es)
WO (1) WO2016130589A2 (es)

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