Target point, preparation and method for treating human ADSL deficiency

Abstract: The present invention discloses target point, preparation and method for treating human ADSL deficiency. The present invention established an ADSL deficiency retrieval model in nematode through RNA interference technique, and found that the sole RNA interference with PAICS gene could increase the expression of ADSL gene. Based on the conclusion of nematode model study, it can be foreseen that through interference with PAICS gene expression in ADSL deficient patient, accumulation of SAICAR in patient body fluid will be decreased and thereby reducing damage to cells, achieving the object of treating human ADSL deficiency or relieving symptoms.
Patent Number: US2016369281(A1)
Public Date:
Inventor: HE XIONGLEI [CN]
Application Number: US201514741158 20150616Global Dossier
Application Date:
Priority Number: US201514741158 20150616
International Classification: C12N15/113
Cooperative Classification: C12N15/1137; C12Y401/01021; C12Y403/02002; C12Y603/02006; C12N2310/14