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Application filed by Bamboo Therapeutics Inc, Univ North Carolina Chapel HillfiledCriticalBamboo Therapeutics Inc
Publication of AR108848A1publicationCriticalpatent/AR108848A1/en
Pharmaceuticals Containing Other Organic And Inorganic Compounds
(AREA)
Medicines That Contain Protein Lipid Enzymes And Other Medicines
(AREA)
Abstract
Esta solicitud se refiere a polinucleótidos que codifican las proteínas de mini-distrofina, vectores virales que comprenden las mismas y métodos para usar las mismas para administración de mini-distrofina a una célula o a un sujeto. Reivindicación 1: Un polinucleótido que codifica una proteína mini-distrofina, dicho polinucleótido comprende: (a) la secuencia de nucleótidos de SEQ ID Nº 1 o una secuencia de nucleótidos por lo menos aproximadamente 90% idéntica a la misma; (b) la secuencia de nucleótidos de SEQ ID Nº 2 o una secuencia de nucleótidos por lo menos aproximadamente 90% idéntica a la misma; o (c) la secuencia de nucleótidos de SEQ ID Nº 3 o una secuencia de nucleótidos por lo menos aproximadamente 90% idéntica a la misma.This application relates to polynucleotides encoding mini-dystrophin proteins, viral vectors comprising the same and methods for using them for administration of mini-dystrophin to a cell or to a subject. Claim 1: A polynucleotide encoding a mini-dystrophin protein, said polynucleotide comprises: (a) the nucleotide sequence of SEQ ID No. 1 or a nucleotide sequence at least about 90% identical thereto; (b) the nucleotide sequence of SEQ ID No. 2 or a nucleotide sequence at least about 90% identical thereto; or (c) the nucleotide sequence of SEQ ID No. 3 or a nucleotide sequence at least about 90% identical thereto.
ARP170101711A2016-06-212017-06-21
OPTIMIZED MINI-DISTROPHIN GENES AND EXPRESSION CASSETTES AND THEIR USE
AR108848A1
(en)
Prevotella and francisella crispr protein 1 (cpf1) isolated from acidaminococcus sp. bv3l6; fusion protein comprising it; nucleic acid; vector; methods for altering the genome of a cell or a double-stranded DNA molecule in vitro using such proteins (divisional de sol. 201903004)
Aav / upr-plus virus, upr-plus fusion protein, genetic treatment method and its use in the treatment of neurodegenerative diseases, such as parkinson's and huntington diseases, among others.