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MX2018007330A - Vectores virales adeno-asociados para tratar mucolipidosis de tipo ii. - Google Patents

Vectores virales adeno-asociados para tratar mucolipidosis de tipo ii.

Info

Publication number
MX2018007330A
MX2018007330A MX2018007330A MX2018007330A MX2018007330A MX 2018007330 A MX2018007330 A MX 2018007330A MX 2018007330 A MX2018007330 A MX 2018007330A MX 2018007330 A MX2018007330 A MX 2018007330A MX 2018007330 A MX2018007330 A MX 2018007330A
Authority
MX
Mexico
Prior art keywords
mucolipidosis type
iii
vectors
raav
adeno
Prior art date
Application number
MX2018007330A
Other languages
English (en)
Inventor
Yew Nelson
Jin Dong-Kyu
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.)
Filing date
Publication date
Application filed by Genzyme Corp filed Critical Genzyme Corp
Publication of MX2018007330A publication Critical patent/MX2018007330A/es

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    • C12N15/79—Vectors or expression systems specially adapted for eukaryotic hosts
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Abstract

En la presente se proporcionan vectores de virus adeno-asociado recombinante (rAAV) que comprenden ácido nucleico que codifica N-acetilglucosamina-1-fosfato transferasa, subunidades alfa y beta (GNPTAB) y al menos una repetición terminal invertida (1TR) de AAV. En algunas modalidades, los vectores de rAAV pueden estar incluidos en una partícula de rAAV, que puede estar contenida en composiciones farmacéuticas y kits. Estos vectores, partículas, composiciones, y kits pueden encontrar uso, inter anal en métodos y usos relacionados con el tratamiento de mucolipidosis tipo II (ML II) o mucolipidosis tipo Ill (ML III) en un mamífero, o relacionados con el incremento del tamalio corporal, contenido mineral Óseo, y/o densidad mineral Ósea en un mamífero con mucolipidosis tipo II (ML II) o mucolipidosis tipo III (ML III).
MX2018007330A 2015-12-15 2016-12-14 Vectores virales adeno-asociados para tratar mucolipidosis de tipo ii. MX2018007330A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201562267502P 2015-12-15 2015-12-15
PCT/US2016/066611 WO2017106313A1 (en) 2015-12-15 2016-12-14 Adeno-associated viral vectors for treating mucolipidosis type ii

Publications (1)

Publication Number Publication Date
MX2018007330A true MX2018007330A (es) 2019-03-14

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MX2018007330A MX2018007330A (es) 2015-12-15 2016-12-14 Vectores virales adeno-asociados para tratar mucolipidosis de tipo ii.

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US (1) US20200261598A1 (es)
EP (1) EP3390644B1 (es)
JP (3) JP7347932B2 (es)
KR (1) KR102833376B1 (es)
CN (1) CN108603199A (es)
AU (2) AU2016372035B2 (es)
CA (1) CA3008268A1 (es)
ES (1) ES2991277T3 (es)
IL (1) IL259964B (es)
MX (1) MX2018007330A (es)
RU (1) RU2742612C2 (es)
SG (2) SG11201804994QA (es)
WO (1) WO2017106313A1 (es)

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CA3171166A1 (en) 2020-02-13 2021-08-19 Tenaya Therapeutics, Inc. Gene therapy vectors for treating heart disease

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AU2016372035B2 (en) 2023-03-30
KR102833376B1 (ko) 2025-07-11
AU2016372035A1 (en) 2018-08-02
CA3008268A1 (en) 2017-06-22
WO2017106313A1 (en) 2017-06-22
CN108603199A (zh) 2018-09-28
SG10201912935WA (en) 2020-02-27
IL259964A (en) 2018-07-31
EP3390644B1 (en) 2024-07-31
ES2991277T3 (es) 2024-12-03
IL259964B (en) 2022-09-01
EP3390644A1 (en) 2018-10-24
BR112018012015A2 (pt) 2018-12-04
US20200261598A1 (en) 2020-08-20
KR20180098294A (ko) 2018-09-03
JP7347932B2 (ja) 2023-09-20
JP2019500034A (ja) 2019-01-10
SG11201804994QA (en) 2018-07-30
JP2024056832A (ja) 2024-04-23
JP2022046635A (ja) 2022-03-23
RU2742612C2 (ru) 2021-02-09
AU2023204116A1 (en) 2023-07-13
RU2018124448A3 (es) 2020-08-14
RU2018124448A (ru) 2020-01-17

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