PE20211114A1 - Metodos y composiciones para terapia con celulas oculares - Google Patents

Metodos y composiciones para terapia con celulas oculares

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Publication number
PE20211114A1
PE20211114A1 PE2021000558A PE2021000558A PE20211114A1 PE 20211114 A1 PE20211114 A1 PE 20211114A1 PE 2021000558 A PE2021000558 A PE 2021000558A PE 2021000558 A PE2021000558 A PE 2021000558A PE 20211114 A1 PE20211114 A1 PE 20211114A1
Authority
PE
Peru
Prior art keywords
limbar
modified
reduced
stem cell
compositions
Prior art date
Application number
PE2021000558A
Other languages
English (en)
Inventor
Frada Berenshteyn
Bo Han
Xueshi Hao
Jessica Heyder
Timothy Z Hoffman
Qihui Jin
Arnaud Lacoste
Jun Liu
Yahu Liu
Tingting Mo
Bradley Andrew Murray
Daniel Joseph O'connell
Jianfeng Pan
Yun Feng Xie
Shanshan Yan
Yefen Zou
Original Assignee
Novartis Ag
Intellia Therapeutics 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 Novartis Ag, Intellia Therapeutics Inc filed Critical Novartis Ag
Publication of PE20211114A1 publication Critical patent/PE20211114A1/es

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    • C12N5/06Animal cells or tissues; Human cells or tissues
    • C12N5/0602Vertebrate cells
    • C12N5/0618Cells of the nervous system
    • C12N5/0623Stem cells
    • AHUMAN NECESSITIES
    • A61MEDICAL OR VETERINARY SCIENCE; HYGIENE
    • A61KPREPARATIONS FOR MEDICAL, DENTAL OR TOILETRY PURPOSES
    • A61K31/00Medicinal preparations containing organic active ingredients
    • A61K31/33Heterocyclic compounds
    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/435Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/4353Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems
    • A61K31/4375Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom ortho- or peri-condensed with heterocyclic ring systems the heterocyclic ring system containing a six-membered ring having nitrogen as a ring heteroatom, e.g. quinolizines, naphthyridines, berberine, vincamine
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    • A61K31/435Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with one nitrogen as the only ring hetero atom
    • A61K31/47Quinolines; Isoquinolines
    • A61K31/472Non-condensed isoquinolines, e.g. papaverine
    • A61K31/4725Non-condensed isoquinolines, e.g. papaverine containing further heterocyclic rings
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    • A61K31/395Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins
    • A61K31/495Heterocyclic compounds having nitrogen as a ring hetero atom, e.g. guanethidine or rifamycins having six-membered rings with two or more nitrogen atoms as the only ring heteroatoms, e.g. piperazine or tetrazines
    • A61K31/505Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim
    • A61K31/519Pyrimidines; Hydrogenated pyrimidines, e.g. trimethoprim ortho- or peri-condensed with heterocyclic rings
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    • A61K35/12Materials from mammals; Compositions comprising non-specified tissues or cells; Compositions comprising non-embryonic stem cells; Genetically modified cells
    • A61K35/30Nerves; Brain; Eyes; Corneal cells; Cerebrospinal fluid; Neuronal stem cells; Neuronal precursor cells; Glial cells; Oligodendrocytes; Schwann cells; Astroglia; Astrocytes; Choroid plexus; Spinal cord tissue
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    • C12N9/14Hydrolases (3)
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    • C12N9/22Ribonucleases RNAses, DNAses
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    • C12N2310/00Structure or type of the nucleic acid
    • C12N2310/10Type of nucleic acid
    • C12N2310/20Type of nucleic acid involving clustered regularly interspaced short palindromic repeats [CRISPRs]
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    • C12N2502/08Coculture with; Conditioned medium produced by cells of the nervous system
    • C12N2502/085Coculture with; Conditioned medium produced by cells of the nervous system eye cells
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    • C12N2800/80Vectors containing sites for inducing double-stranded breaks, e.g. meganuclease restriction sites

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  • Nuclear Medicine, Radiotherapy & Molecular Imaging (AREA)
  • Dermatology (AREA)
  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Pharmaceuticals Containing Other Organic And Inorganic Compounds (AREA)
  • Micro-Organisms Or Cultivation Processes Thereof (AREA)

Abstract

SE REFIERE A UNA CELULA MADRE LIMBAR MODIFICADA, QUE HA REDUCIDO O ELIMINADO LA EXPRESION DE BETA-2-MICROGLOBULINA (B2M) EN RELACION CON UNA CELULA MADRE LIMBAR NO MODIFICADA, EN DONDE LA EXPRESION DE B2M SE REDUCE O ELIMINA POR UN SISTEMA CRISPR QUE COMPRENDE UNA MOLECULA DE ARNg QUE COMPRENDE UN DOMINIO DE DIRECCIONAMIENTO COMPLEMENTARIO A UNA SECUENCIA DIANA EN EL GEN B2M, EN DONDE LA REGION DIANA DE LA MOLECULA DE ARNg PARA B2M COMPRENDE UN DOMINIO DE DIRECCIONAMIENTO QUE COMPRENDE LA SECUENCIA DE CUALQUIERA DE LAS SEQ ID NOs: 23-105 O 108-119 O 134-140. TAMBIEN SE REFIERE A UN METODO DE PREPARACION DE UNA CELULA MADRE LIMBAR MODIFICADA O POBLACION DE CELULAS MADRE LIMBARES MODIFICADAS, Y A UNA COMPOSICION QUE COMPRENDE LAS MISMAS, LA CUAL ES UTIL EN EL TRATAMIENTO DE UNA ENFERMEDAD OCULAR TAL COMO DEFICIENCIA DE CELULAS MADRE LIMBARES.
PE2021000558A 2018-10-26 2019-10-25 Metodos y composiciones para terapia con celulas oculares PE20211114A1 (es)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201862750962P 2018-10-26 2018-10-26
US201962902639P 2019-09-19 2019-09-19
PCT/IB2019/059162 WO2020084580A1 (en) 2018-10-26 2019-10-25 Methods and compositions for ocular cell therapy

Publications (1)

Publication Number Publication Date
PE20211114A1 true PE20211114A1 (es) 2021-06-22

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PE2021000558A PE20211114A1 (es) 2018-10-26 2019-10-25 Metodos y composiciones para terapia con celulas oculares

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US (1) US20200131474A1 (es)
EP (1) EP3870289A1 (es)
JP (1) JP2022505658A (es)
KR (1) KR20210069075A (es)
CN (1) CN112969472A (es)
AU (1) AU2019365590A1 (es)
BR (1) BR112021007628A2 (es)
CA (1) CA3116512A1 (es)
CL (1) CL2021001034A1 (es)
CO (1) CO2021005289A2 (es)
CR (1) CR20210196A (es)
CU (1) CU20210033A7 (es)
EC (1) ECSP21028556A (es)
IL (1) IL282447A (es)
JO (1) JOP20210080A1 (es)
MX (1) MX2021004532A (es)
PE (1) PE20211114A1 (es)
PH (1) PH12021550761A1 (es)
SG (1) SG11202102615RA (es)
TW (1) TW202030324A (es)
UY (1) UY38427A (es)
WO (1) WO2020084580A1 (es)

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CN115671398B (zh) * 2022-11-22 2024-03-01 首都医科大学附属北京同仁医院 一种3d打印仿生角膜缘移植物及其制备方法和用途

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