Porphobilinogen deaminase gene therapy

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United States of America Patent

PATENT NO 8697665
SERIAL NO

13121532

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ATTORNEY / AGENT: (SPONSORED)

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Abstract

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The present invention relates to nucleotide sequences coding for human porphobilinogen deaminase that are optimised for higher expression in mammalian cells. The invention further relates to DNA constructs comprising such optimised synthetic coding sequences for use in gene therapy of conditions caused by a deficiency in porphobilinogen deaminase, such as acute intermittent porphyria. Accordingly, the present invention relates to a nucleic acid or a nucleic acid construct comprising a nucleotide sequence coding for a human porphobilinogen deaminase, wherein at least 320 of the codons coding for the human porphobilinogen deaminase are identical to the codons in SEQ ID NO: 1 or wherein at least 305 of the codons coding for the human porphobilinogen deaminase are identical to the codons in SEQ ID NO: 3.

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Patent Owner(s)

Patent OwnerAddress
UNIQURE IP B VAMSTERDAM

International Classification(s)

Inventor(s)

Inventor Name Address # of filed Patents Total Citations
Fontanellas, Romá Antonio Pamplona Navarra, ES 1 32
González, Aseguinolaza Gloria Pamplona Navarra, ES 6 39
Pañeda, Rodriguez Maria Astrid Pamplona Navarra, ES 1 32
Petry, Harald Amsterdam, NL 9 95
Prieto, Valtueña Jesús Maria Pamplona Navarra, ES 5 42
Rodriguez, Pena Maria Sol Le Mont-sur-Lausanne, CH 2 32
Twisk, Jaap Amsterdam, NL 2 32
Van, Deventer Sander Jan Hendrik Amsterdam, NL 11 34

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