Ｘ連鎖性網膜色素変性症の治療のためのＲＰ２及びＲＰＧＲベクター

Title:

Ｘ連鎖性網膜色素変性症の治療のためのＲＰ２及びＲＰＧＲベクター

Document Type and Number:

Japanese Patent JP6935049

Kind Code:

B2

Abstract:

Disclosed are adeno-associated virus (AAV) vectors comprising a nucleotide sequence encoding RP2 or RPGR-ORF15 and related pharmaceutical compositions. Also disclosed are methods of treating or preventing X-linked retinitis pigmentosa, increasing photoreceptor number in a retina of a mammal, and increasing visual acuity of a mammal using the vectors and pharmaceutical compositions.

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Inventors:

Wu, Jijan
Swallow, Anand
McCarthy, Sud Hazil
Hiryanna, Suger

Application Number:

JP2017547425A

Publication Date:

September 15, 2021

Filing Date:

March 11, 2016

Export Citation:

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Assignee:

THE UNITED STATES OF AMERICA

International Classes:

C12N15/864; A61K9/10; A61K35/76; A61K48/00; A61P27/02

Domestic Patent References:

JP2014517694A
JP2001500014A

Foreign References:

WO2014011210A1

Other References:

MOOKHERJEE, Suddhasil et al.,A Long-Term Study of AAV Vector-Mediated Gene Therapy for X-linked Retinitis Pigmentosa (XLRP) Due to RP2 Mutation,Molecular Therapy,2014年 5月 1日,vol. 22, supplement 1,p. S46
STRAUSBERG, R.L. et al.,Definition: Retinitis pigmentosa 2 (X-linked recessive) [Homo sapiens].,Database GenBank, [online], Accession No. AAH43348, , 15-JUL-2006 uploaded

Attorney, Agent or Firm:

Takashima Ichi
Mitsunori Kamata
Kyoko Doi
Yaeko Tamura
Hirofumi Toma
Atsuko Akai
Tomiya Tozaki

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