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Application filed by Amicus Therapeutics IncfiledCriticalAmicus Therapeutics Inc
Publication of AR124827A1publicationCriticalpatent/AR124827A1/en
Micro-Organisms Or Cultivation Processes Thereof
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Pharmaceuticals Containing Other Organic And Inorganic Compounds
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Medicines That Contain Protein Lipid Enzymes And Other Medicines
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Abstract
La presente divulgación se refiere al suministro por virus adeno-asociado recombinante (rAAV) de un polinucleótido de lipofuscinosis neuronal ceroidea 3 (CLN3). La divulgación provee rAAV y métodos para usar el rAAV para la terapia génica de CLN3 de la lipofuscinosis neuronal ceroidea o la enfermedad de CLN3-Batten.The present disclosure relates to recombinant adeno-associated virus (rAAV) delivery of a neuronal ceroid lipofuscinosis 3 (CLN3) polynucleotide. The disclosure provides rAAV and methods of using rAAV for CLN3 gene therapy of neuronal ceroid lipofuscinosis or CLN3-Batten disease.
ARP220100241A2021-02-052022-02-07
DELIVERY BY ADENO-ASSOCIATED VIRUS OF POLYNUCLEOTIDE CLN3
AR124827A1
(en)
COMPOUND, PHARMACEUTICAL COMPOSITION AND METHODS FOR INHIBITING SOS1 IN A SUBJECT, FOR INHIBITING THE INTERACTION OF SOS1 AND A PROTEIN, TO TREAT OR PREVENT A DISEASE AND TO TREAT OR PREVENT CANCER
Electroacupuncture alleviates nerve injury after cerebra ischemia in rats through inhibiting cell apoptosis and changing the balance of MMP-9/TIMP-1 expression