C - Chemistry – Metallurgy – 12 – N
Patent
C - Chemistry, Metallurgy
12
N
C12N 15/12 (2006.01) A61K 31/70 (2006.01) A61K 39/395 (2006.01) C07K 14/47 (2006.01) C07K 16/18 (2006.01) C12N 9/10 (2006.01) C12N 15/11 (2006.01) C12Q 1/68 (2006.01) G01N 33/566 (2006.01) A61K 38/00 (2006.01)
Patent
CA 2087739
ABSTRACT Point mutations and/ or deletions in the human hypoxanthine- guanine phosphoribosyl transferase (HPRT) gene is believed to be the cause of Lesch-Nyhan syndrome in humans; however, the disastrous neurological symptoms which are characteristic of this disease cannot be easily explained by these mutations/deletions or even by the complete loss of the gene. There is no known cure for the disease. We reconstructed a cDNA (chprt) which is 100% homologous to a region of the antisense strand of hprt mRNA. The cDNA encodes a 30 amino acid protein (phrpt) which appears to be an intracellular activator of proteins involved in human neurological diseases. Because of the antisense relation with hprt mRNA, phprt cannot be expressed in humans carrying a normal wild type hprt gene; however, mutations in critical regions of the hprt gene can stop antisense repression and permit expression of phprt and the appearance of the pathophysiological symptoms of Lesch-Nyhan disease. The antisense protein is a very good target for immunotherapeutic reagents to stop the devastating neurological processes caused in humans afflicted with Lesch-Nyhan.
Bergmann Johanna E.
Preddie Rick E.
Bergmann Johanna E.
Na
Preddie Rick E.
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