Compositions and methods for treating leber's hereditary optic neuropathy

A technology for compositions and pharmaceutical compounds, which can be applied in the fields of botanical equipment and methods, biochemical equipment and methods, and introduction of foreign genetic material using vectors, which can solve problems such as the reduction of mitochondrial NADH dehydrogenase activity
CN112584874APending Publication Date: 2021-03-30WUHAN NEUROPHTH BIOTECHNOLOGY LTD CO
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Patent Information

Authority / Receiving Office
CN ยท China
Current Assignee / Owner
WUHAN NEUROPHTH BIOTECHNOLOGY LTD CO
Publication Date
2021-03-30

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Abstract

Provided is a recombinant nucleic acid, comprising: a mitochondrial targeting sequence; a mitochondrial protein coding sequence, wherein the mitochondrial protein coding sequence encodes a polypeptidecomprising a mitochondrial protein; and a 3'UTR nucleic acid sequence. Also provided are a pharmaceutical composition comprising the recombinant nucleic acid and a method of treating Leber's hereditary optic neuropathy (LHON) using the pharmaceutical composition.
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Description

[0001] Cross-reference

[0002] The present application claims PCT / CN2018 / 103937 PCT applications submitted on September 4, 2018; PCT / CN2018 / 113799, filed on November 2, 2018; CN201811230856.2, October 22, 2018 Application; PCT / CN2018 / 118662 submitted on November 30, 2018; China Application for CN20181221305.x, on October 19, 2018; PCT / CN2019 / 070461 submitted on January 4, 2018 PCT application The equity of China's application is submitted by CN201810948193.1, filed on August 20, 2018; each of the above applications is incorporated herein by reference.

[0003] Quote for sequence list

[0004] The present application contains sequence lists that have been incorporated herein by the EFS-Web in ASCII format and referenced in the reference. The ASCII copy founded on August 16, 2019 named WNBT-005_02WO_ST25.TXT, and the size is 298KB. Background technique

[0005] Leber's Hereditary Neuropathy, LHON is a mitochondrial genetic (RGC) and its axial derrotharily derived vap...

Claims

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