KR20240164969A - Nt-3 유전자 치료를 위한 방법 및 물질 - Google Patents

Nt-3 유전자 치료를 위한 방법 및 물질 Download PDF

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KR20240164969A
KR20240164969A KR1020247037712A KR20247037712A KR20240164969A KR 20240164969 A KR20240164969 A KR 20240164969A KR 1020247037712 A KR1020247037712 A KR 1020247037712A KR 20247037712 A KR20247037712 A KR 20247037712A KR 20240164969 A KR20240164969 A KR 20240164969A
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aav
muscle
raav
neuropathy
tmck
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자리페 사헨크
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더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈
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    • C12N15/09—Recombinant DNA-technology
    • 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
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    • A61K38/18—Growth factors; Growth regulators
    • A61K38/185—Nerve growth factor [NGF]; Brain derived neurotrophic factor [BDNF]; Ciliary neurotrophic factor [CNTF]; Glial derived neurotrophic factor [GDNF]; Neurotrophins, e.g. NT-3
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    • A61K48/0058—Nucleic acids adapted for tissue specific expression, e.g. having tissue specific promoters as part of a contruct
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    • A—HUMAN NECESSITIES
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    • C07K14/435—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
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    • C12N2750/00011—Details
    • C12N2750/14011—Parvoviridae
    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
    • C12N2750/14141—Use of virus, viral particle or viral elements as a vector
    • C12N2750/14143—Use of virus, viral particle or viral elements as a vector viral genome or elements thereof as genetic vector
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    • C12N2800/00—Nucleic acids vectors
    • C12N2800/10—Plasmid DNA
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    • C12N2830/00—Vector systems having a special element relevant for transcription
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    • C12N2830/00—Vector systems having a special element relevant for transcription
    • C12N2830/50—Vector systems having a special element relevant for transcription regulating RNA stability, not being an intron, e.g. poly A signal
    • Y—GENERAL TAGGING OF NEW TECHNOLOGICAL DEVELOPMENTS; GENERAL TAGGING OF CROSS-SECTIONAL TECHNOLOGIES SPANNING OVER SEVERAL SECTIONS OF THE IPC; TECHNICAL SUBJECTS COVERED BY FORMER USPC CROSS-REFERENCE ART COLLECTIONS [XRACs] AND DIGESTS
    • Y02—TECHNOLOGIES OR APPLICATIONS FOR MITIGATION OR ADAPTATION AGAINST CLIMATE CHANGE
    • Y02A—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE
    • Y02A50/00—TECHNOLOGIES FOR ADAPTATION TO CLIMATE CHANGE in human health protection, e.g. against extreme weather
    • Y02A50/30—Against vector-borne diseases, e.g. mosquito-borne, fly-borne, tick-borne or waterborne diseases whose impact is exacerbated by climate change

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KR1020247037712A 2017-10-20 2018-10-19 Nt-3 유전자 치료를 위한 방법 및 물질 Pending KR20240164969A (ko)

Applications Claiming Priority (8)

Application Number Priority Date Filing Date Title
US201762574828P 2017-10-20 2017-10-20
US62/574,828 2017-10-20
US201862676687P 2018-05-25 2018-05-25
US62/676,687 2018-05-25
US201862741335P 2018-10-04 2018-10-04
US62/741,335 2018-10-04
KR1020207014064A KR20200083495A (ko) 2017-10-20 2018-10-19 Nt-3 유전자 치료를 위한 방법 및 물질
PCT/US2018/056765 WO2019079755A1 (en) 2017-10-20 2018-10-19 METHODS AND MATERIALS FOR NT-3 GENE THERAPY

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KR1020207014064A Division KR20200083495A (ko) 2017-10-20 2018-10-19 Nt-3 유전자 치료를 위한 방법 및 물질

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KR20240164969A true KR20240164969A (ko) 2024-11-21

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KR1020247037712A Pending KR20240164969A (ko) 2017-10-20 2018-10-19 Nt-3 유전자 치료를 위한 방법 및 물질
KR1020207014064A Ceased KR20200083495A (ko) 2017-10-20 2018-10-19 Nt-3 유전자 치료를 위한 방법 및 물질

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US (2) US11926653B2 (enExample)
EP (1) EP3697916A4 (enExample)
JP (3) JP2021500029A (enExample)
KR (2) KR20240164969A (enExample)
CN (2) CN119193705A (enExample)
AU (2) AU2018351528B2 (enExample)
BR (1) BR112020007790A2 (enExample)
CA (1) CA3079416A1 (enExample)
CL (1) CL2020001060A1 (enExample)
CO (1) CO2020006148A2 (enExample)
IL (2) IL321310A (enExample)
MA (1) MA50412A (enExample)
MX (2) MX2020004035A (enExample)
MY (1) MY207563A (enExample)
SA (1) SA520411798B1 (enExample)
SG (1) SG11202003285PA (enExample)
TW (2) TWI879721B (enExample)
WO (1) WO2019079755A1 (enExample)

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KR20210028162A (ko) 2018-06-29 2021-03-11 더 리서치 인스티튜트 앳 네이션와이드 칠드런스 하스피탈 지대근 이영양증 2a형을 치료하기 위한 재조합 아데노 연관 바이러스 생성물 및 방법
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EP4025258A4 (en) * 2019-09-03 2023-09-06 University of Cincinnati METHODS AND COMPOSITIONS FOR THE TREATMENT OF AMYOTROPHIC LATERAL SCLEROSIS
WO2021211705A1 (en) * 2020-04-14 2021-10-21 Research Institute At Nationwide Children's Hospital Treatment of sarcopenia using nt-3 gene therapy
WO2021211713A1 (en) * 2020-04-14 2021-10-21 Research Institute At Nationwide Children's Hospital Treatment of charcot-marie-tooth axonal type 2d using nt-3 gene therapy
CN116440252A (zh) * 2020-06-16 2023-07-18 温州医科大学附属第二医院（温州医科大学附属育英儿童医院） 神经营养因子3在制备提高睾丸中睾酮含量的药物中的应用
EP4108263A3 (en) * 2021-06-02 2023-03-22 Research Institute at Nationwide Children's Hospital Recombinant adeno-associated virus products and methods for treating limb girdle muscular dystrophy 2a
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CN115838725B (zh) * 2022-12-30 2023-09-08 广州派真生物技术有限公司 在哺乳动物心脏中特异性启动基因的启动子序列及其应用
AU2024265705A1 (en) 2023-05-02 2025-11-20 Research Institute At Nationwide Children's Hospital Gene therapy for treatment of protein misfolding diseases
WO2024229115A1 (en) * 2023-05-02 2024-11-07 Research Institute At Nationwide Children's Hospital Treatment of multiple sclerosis using nt-3 gene therapy
CN119899809A (zh) * 2025-01-07 2025-04-29 广州派真生物技术有限公司 一种提高重组腺相关病毒生产效率的方法及应用

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