Novel Fragile X Constructs
AssigneeuniQure biopharma B.V.
InventorsLodewijk Julius Anton Toonen, Xu Tao, Astrid Valles-Sanchez
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Abstract
The present invention relates to a nucleic acid construct comprising a nucleotide sequence encoding two or more Fragile X Messenger Ribonucleoprotein (FMRP) isoforms, which also finds application in the development of AAV vectors that lead to the successful (in vivo) expression of two or more FMRP isoforms and the beneficial results which are produced by the application of such therapy in terms of improved cognitive and/or behavioural symptoms of Fragile-X Syndrome.
Claims20
A nucleic acid construct comprising a nucleotide sequence encoding two or more Fragile X Messenger Ribonucleoprotein (FMRP) isoforms, wherein the nucleic acid comprises a first artificial intron between exons 16 and 17.
-17. (canceled)
A method for treating of a trinucleotide repeat disorder in a subject in need thereof, the method comprising administering the nucleic acid construct according to the subject.
Dependencies are inferred from the claim text.