OPTIMIZED CLN1 GENES AND EXPRESSION CASSETTES AND THEIR USE
作者： Steven Gray
DOI:
关键词: Computational biology 、 Biology 、 Fragment (computer graphics) 、 Open reading frame 、 Nucleic acid sequence 、 Batten disease 、 Polynucleotide 、 Gene 、 PPT1
摘要: This invention relates to polynucleotides comprising a nucleotide sequence encoding PPT1 polypeptide or fragment thereof, vectors (viral non-viral vectors) the same, and methods of using same for delivery open reading frame cell subject treat infantile neuronal lipofuscinosis (infantile Batten disease). The comprise an optimized CLN1 frame.
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