JOP20190200A1 - تركيبات نافعة في معالجة ضمور العضل النخاعي - Google Patents

تركيبات نافعة في معالجة ضمور العضل النخاعي

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Publication number
JOP20190200A1
JOP20190200A1 JOP/2019/0200A JOP20190200A JOP20190200A1 JO P20190200 A1 JOP20190200 A1 JO P20190200A1 JO P20190200 A JOP20190200 A JO P20190200A JO P20190200 A1 JOP20190200 A1 JO P20190200A1
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JO
Jordan
Prior art keywords
muscular atrophy
spinal muscular
treatment
compositions useful
smn
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Application number
JOP/2019/0200A
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English (en)
Inventor
James M Wilson
Nathan Katz
Christian Hinderer
Qiang Wang
Original Assignee
Univ Pennsylvania
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Publication date
Application filed by Univ Pennsylvania filed Critical Univ Pennsylvania
Publication of JOP20190200A1 publication Critical patent/JOP20190200A1/ar

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    • A—HUMAN NECESSITIES
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    • C12N15/63—Introduction of foreign genetic material using vectors; Vectors; Use of hosts therefor; Regulation of expression
    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
    • C12N15/85—Vectors or expression systems specially adapted for eukaryotic hosts for animal cells
    • C12N15/86—Viral vectors
    • C12N15/864—Parvoviral vectors, e.g. parvovirus, densovirus
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
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    • A01K2227/10—Mammal
    • A01K2227/105—Murine
    • A—HUMAN NECESSITIES
    • A01—AGRICULTURE; FORESTRY; ANIMAL HUSBANDRY; HUNTING; TRAPPING; FISHING
    • A01K—ANIMAL HUSBANDRY; AVICULTURE; APICULTURE; PISCICULTURE; FISHING; REARING OR BREEDING ANIMALS, NOT OTHERWISE PROVIDED FOR; NEW BREEDS OF ANIMALS
    • A01K2267/00—Animals characterised by purpose
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/22—Vectors comprising a coding region that has been codon optimised for expression in a respective host

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  • Medicines Containing Material From Animals Or Micro-Organisms (AREA)
  • Medicines That Contain Protein Lipid Enzymes And Other Medicines (AREA)
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Abstract

يتعلق الاختراع الحالي بوصف ناقل rAAV في هذه الوثيقة والذي يتضمن بروتين غلافي AAVhu68 ومجموعة تعبير وراثي واحدة على الأقل في البروتين الغلافي. تشتمل مجموعة التعبير الوراثي الواحدة على الأقل على متواليات حمض نووي تُشفر بروتين SMN الوظيفي ومتواليات تحكم في التعبير الوراثي التي توجه التعبير الوراثي عن المتواليات SMN في خلية عائلة. يتم أيضاً توفير تركيبات تتضمن ناقل rAAVhu68.SMN المذكور وطرق لاستخدامه لمعالجة ضمور العضل النخاعي في مريض.
JOP/2019/0200A 2017-02-28 2017-06-16 تركيبات نافعة في معالجة ضمور العضل النخاعي JOP20190200A1 (ar)

Applications Claiming Priority (3)

Application Number Priority Date Filing Date Title
US201762464756P 2017-02-28 2017-02-28
US201762515902P 2017-06-06 2017-06-06
US201862618437P 2018-01-17 2018-01-17

Publications (1)

Publication Number Publication Date
JOP20190200A1 true JOP20190200A1 (ar) 2019-08-27

Family

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Family Applications (1)

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JOP/2019/0200A JOP20190200A1 (ar) 2017-02-28 2017-06-16 تركيبات نافعة في معالجة ضمور العضل النخاعي

Country Status (19)

Country Link
US (2) US11578341B2 (ar)
EP (1) EP3589739A2 (ar)
JP (2) JP7037574B2 (ar)
KR (1) KR102697086B1 (ar)
CN (1) CN110741089B (ar)
AU (1) AU2018227442B2 (ar)
BR (1) BR112019016444A2 (ar)
CA (1) CA3055619A1 (ar)
CL (1) CL2019002473A1 (ar)
CO (1) CO2019009522A2 (ar)
IL (1) IL268519B2 (ar)
JO (1) JOP20190200A1 (ar)
MA (1) MA47688A (ar)
MX (1) MX2019010276A (ar)
PE (1) PE20191261A1 (ar)
PH (1) PH12019550149A1 (ar)
SA (1) SA519402566B1 (ar)
SG (2) SG10201912495SA (ar)
WO (1) WO2018160585A2 (ar)

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