MX2020003176A - Variantes de capsides de virus adenoasociados y metodos de uso de estas. - Google Patents

Variantes de capsides de virus adenoasociados y metodos de uso de estas.

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Publication number
MX2020003176A
MX2020003176A MX2020003176A MX2020003176A MX2020003176A MX 2020003176 A MX2020003176 A MX 2020003176A MX 2020003176 A MX2020003176 A MX 2020003176A MX 2020003176 A MX2020003176 A MX 2020003176A MX 2020003176 A MX2020003176 A MX 2020003176A
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MX
Mexico
Prior art keywords
aav
methods
virions
muscle cells
associated virus
Prior art date
Application number
MX2020003176A
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English (en)
Inventor
David H Kirn
Melissa Kotterman
David Schaffer
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4D Molecular Therapeutics Inc
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Publication date
Application filed by 4D Molecular Therapeutics Inc filed Critical 4D Molecular Therapeutics Inc
Publication of MX2020003176A publication Critical patent/MX2020003176A/es

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    • A61K38/17—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans
    • A61K38/1703—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates
    • A61K38/1709—Peptides having more than 20 amino acids; Gastrins; Somatostatins; Melanotropins; Derivatives thereof from animals; from humans from vertebrates from mammals
    • A61K38/1719—Muscle proteins, e.g. myosin or actin
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    • C12N2750/14111—Dependovirus, e.g. adenoassociated viruses
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Abstract

En la presente se proporcionan proteínas de cápsides variantes de virus adenoasociados (AAV) que tienen una o más modificaciones en la secuencia de aminoácidos en relación con una proteína de la cápside de AAV parental, que, cuando están presentes en un virión de AAV, confieren un aumento en la infectividad de uno o más tipos de células musculares en comparación con la infectividad de las células musculares por un virión de AAV que comprende la proteína de la cápside de AAV parental no modificada. También se proporcionan viriones de AAV recombinantes y composiciones farmacéuticas de estos que comprenden una proteína de variante de cápside de AAV como se describe en la presente, métodos para producir estos viriones y proteínas de la cápside de rAAV, y métodos para usar estos viriones y proteínas de la cápside de rAAV en la investigación y en la práctica clínica, por ejemplo, en la administración de secuencias de ácidos nucleicos a una o más células musculares para el tratamiento de trastornos y enfermedades musculares.
MX2020003176A 2017-09-20 2018-09-19 Variantes de capsides de virus adenoasociados y metodos de uso de estas. MX2020003176A (es)

Applications Claiming Priority (2)

Application Number Priority Date Filing Date Title
US201762560901P 2017-09-20 2017-09-20
PCT/US2018/051812 WO2019060454A2 (en) 2017-09-20 2018-09-19 CAPSID VARIANT ADENO-ASSOCIATED VIRUSES AND METHODS OF USE

Publications (1)

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MX2020003176A true MX2020003176A (es) 2020-08-20

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MX2020003176A MX2020003176A (es) 2017-09-20 2018-09-19 Variantes de capsides de virus adenoasociados y metodos de uso de estas.

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US (4) US11118192B2 (es)
EP (2) EP3684423B1 (es)
JP (1) JP6825167B2 (es)
KR (2) KR20220070075A (es)
CN (1) CN111836649A (es)
AU (1) AU2018337833B2 (es)
BR (1) BR112020005436B1 (es)
CA (1) CA3076348A1 (es)
CL (1) CL2020000728A1 (es)
CO (1) CO2020004738A2 (es)
CR (1) CR20200165A (es)
DK (1) DK3684423T5 (es)
ES (1) ES2948902T3 (es)
FI (1) FI3684423T3 (es)
IL (1) IL273261A (es)
MX (1) MX2020003176A (es)
NZ (1) NZ763018A (es)
PE (1) PE20201264A1 (es)
PH (1) PH12020550094A1 (es)
PT (1) PT3684423T (es)
RU (1) RU2770922C2 (es)
SG (1) SG11202002276VA (es)
UA (1) UA127455C2 (es)
WO (1) WO2019060454A2 (es)
ZA (1) ZA202001679B (es)

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