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1. (WO2017095489) USE OF MICRORNA PRECURSORS AS DRUGS FOR INDUCING CD34-POSITIVE ADULT STEM CELL EXPANSION
Latest bibliographic data on file with the International Bureau

Pub. No.: WO/2017/095489 International Application No.: PCT/US2016/049583
Publication Date: 08.06.2017 International Filing Date: 31.08.2016
Chapter 2 Demand Filed: 02.03.2017
IPC:
C12N 15/113 (2010.01) ,A61K 31/713 (2006.01) ,C12N 5/10 (2006.01) ,C12N 5/074 (2010.01) ,A61P 25/28 (2006.01) ,A61P 39/00 (2006.01)
C CHEMISTRY; METALLURGY
12
BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
N
MICRO-ORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICRO-ORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
15
Mutation or genetic engineering; DNA or RNA concerning genetic engineering, vectors, e.g. plasmids, or their isolation, preparation or purification; Use of hosts therefor
09
Recombinant DNA-technology
11
DNA or RNA fragments; Modified forms thereof
113
Non-coding nucleic acids modulating the expression of genes, e.g. antisense oligonucleotides
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
K
PREPARATIONS FOR MEDICAL, DENTAL, OR TOILET PURPOSES
31
Medicinal preparations containing organic active ingredients
70
Carbohydrates; Sugars; Derivatives thereof
7088
Compounds having three or more nucleosides or nucleotides
713
Double-stranded nucleic acids or oligonucleotides
C CHEMISTRY; METALLURGY
12
BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
N
MICRO-ORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICRO-ORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
5
Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
10
Cells modified by introduction of foreign genetic material, e.g. virus-transformed cells
C CHEMISTRY; METALLURGY
12
BIOCHEMISTRY; BEER; SPIRITS; WINE; VINEGAR; MICROBIOLOGY; ENZYMOLOGY; MUTATION OR GENETIC ENGINEERING
N
MICRO-ORGANISMS OR ENZYMES; COMPOSITIONS THEREOF; PROPAGATING, PRESERVING, OR MAINTAINING MICRO-ORGANISMS; MUTATION OR GENETIC ENGINEERING; CULTURE MEDIA
5
Undifferentiated human, animal or plant cells, e.g. cell lines; Tissues; Cultivation or maintenance thereof; Culture media therefor
07
Animal cells or tissues
071
Vertebrate cells or tissues, e.g. human cells or tissues
074
Adult stem cells
A HUMAN NECESSITIES
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
P
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
25
Drugs for disorders of the nervous system
28
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
61
MEDICAL OR VETERINARY SCIENCE; HYGIENE
P
SPECIFIC THERAPEUTIC ACTIVITY OF CHEMICAL COMPOUNDS OR MEDICINAL PREPARATIONS
39
General protective or antinoxious agents
Applicants:
LIN, Shi-Lung [US/US]; US
CHANG, Donald [US/US]; US
WU, David TS [US/US]; US
Inventors:
LIN, Shi-Lung; US
CHANG, Donald; US
WU, David TS; US
Agent:
MCROBBIE, Craig A.; US
Priority Data:
15/048,96419.02.2016US
15/167,22627.05.2016US
62/262,28002.12.2015US
Title (EN) USE OF MICRORNA PRECURSORS AS DRUGS FOR INDUCING CD34-POSITIVE ADULT STEM CELL EXPANSION
(FR) UTILISATION DE PRÉCURSEURS DE MICRO-ARN COMME MÉDICAMENTS PERMETTANT D'INDUIRE L'EXPANSION DE CELLULES SOUCHES ADULTES
Abstract:
(EN) This invention generally relates to a composition and method for inducing the expansion and/or regeneration of CD34-positive adult stem cell population, which is useful for developing drugs/vaccines and/or therapies against a variety of ageing-related degenerative diseases in humans. Also, the present invention teaches the production and purification methods required for making high quality and high quantity of small hairpin-like RNA (shRNA) compositions, such as microRNA precursors (pri-miRNA and pre-miRNA) and short interfering RNAs (siRNA), which are useful for treating human ageing-related diseases, such as, but not limited, Alzheimer's diseases, Parkinson's diseases, osteoporosis, diabetes, and cancers. The resulting shRNAs, preferably pre-miRNAs, are useful for developing therapeutic drugs against human degenerative diseases, particularly through a mechanism to induce CD34-positive stem cell expansion and/or regeneration.
(FR) Cette invention concerne de manière générale une composition et une méthode permettant d'induire l'expansion et/ou la régénération d'une population de cellules souches adultes positives pour le CD34, qui est utilisable pour développer des médicaments, vaccins et/ou traitements contre diverses maladies dégénératives liées au vieillissement chez l'homme. En outre, la présente invention concerne les procédés de production et de purification nécessaires à la fabrication de grandes quantités de compositions de haute qualité de petits ARN en épingle à cheveux (ARNsh), tels que des précurseurs de microARN (pri-miARN et pré-miARN) et de petits ARN interférents (siARN), qui sont utilisables pour traiter des maladies liées au vieillissement, telles que, sans y être limitées, la maladie d'Alzheimer, la maladie de Parkinson, l'ostéoporose, le diabète et les cancers. Les ARNsh qui en résultent, de préférence des pré-miARN, sont utilisables pour développer des médicaments thérapeutiques contre des maladies dégénératives, en particulier par l'intermédiaire d'un mécanisme qui permet d'induire l'expansion et/ou la régénération de cellules souches positives pour le CD34.
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Designated States: AE, AG, AL, AM, AO, AT, AU, AZ, BA, BB, BG, BH, BN, BR, BW, BY, BZ, CA, CH, CL, CN, CO, CR, CU, CZ, DE, DK, DM, DO, DZ, EC, EE, EG, ES, FI, GB, GD, GE, GH, GM, GT, HN, HR, HU, ID, IL, IN, IR, IS, JP, KE, KG, KN, KP, KR, KZ, LA, LC, LK, LR, LS, LU, LY, MA, MD, ME, MG, MK, MN, MW, MX, MY, MZ, NA, NG, NI, NO, NZ, OM, PA, PE, PG, PH, PL, PT, QA, RO, RS, RU, RW, SA, SC, SD, SE, SG, SK, SL, SM, ST, SV, SY, TH, TJ, TM, TN, TR, TT, TZ, UA, UG, US, UZ, VC, VN, ZA, ZM, ZW
African Regional Intellectual Property Organization (ARIPO) (BW, GH, GM, KE, LR, LS, MW, MZ, NA, RW, SD, SL, ST, SZ, TZ, UG, ZM, ZW)
Eurasian Patent Organization (AM, AZ, BY, KG, KZ, RU, TJ, TM)
European Patent Office (AL, AT, BE, BG, CH, CY, CZ, DE, DK, EE, ES, FI, FR, GB, GR, HR, HU, IE, IS, IT, LT, LU, LV, MC, MK, MT, NL, NO, PL, PT, RO, RS, SE, SI, SK, SM, TR)
African Intellectual Property Organization (BF, BJ, CF, CG, CI, CM, GA, GN, GQ, GW, KM, ML, MR, NE, SN, TD, TG)
Publication Language: English (EN)
Filing Language: English (EN)
Also published as:
CN108431227EP3384026