Abstract:
The present invention provides compounds comprising oligonucleotides complementary to a CLN3 transcript. Certain such compounds are useful for hybridizing to a CLN3 transcript, including but not limited to a CLN3 transcript in a cell. In certain embodiments, such hybridization results in modulation of splicing of the CLN3 transcript. In certain embodiments, such compounds are used to treat one or more symptoms associated with Batten Disease.
Abstract:
The present invention relates generally to the use of erythropoietin (EPO) to facilitate resuscitation from cardiac arrest. For a mammalian subject suffering from cardiac arrest, concurrent administration of EPO with resuscitation after the onset of ventricular fibrillation facilitates the resuscitation. Administration of EPO serves to attenuate myocardial abnormalities caused by cardiac arrest and the resuscitation efforts and favor improved resuscitation outcomes. The main effect of EPO that facilitates resuscitation is the preservation of left ventricular myocardial distensibility leading to improve left ventricular preload and the amount of blood ejected by chest compression. This effect enables higher coronary perfusion pressures to be generated resulting in a higher rate of return of spontaneous circulation and higher survival rates. The very same effect enables the return of spontaneous circulation to occur faster reducing the time a human subject is in cardiac arrest.
Abstract:
The present invention relates generally to the use of γ-glutamyl antioxidants, particularly γ-glutamyl-cysteine, as cytoprotective agents to prevent reperfusion injury (i.e., hemorrhagic transformation) of the blood-brain barrier during reperfusion following an ischemic stroke. The γ-glutamyl antioxidants can be used alone or used in combination with an agent which inhibits the reverse movement of Na/Ca exchange in the blood-brain barrier such as 2-[2-[4-(4-nitrobenzyloxy)phenyl]ethyl]isothiourea methanesulphonate (KB-R7943).
Abstract:
Provided are compounds, methods, and pharmaceutical compositions for modulating the expression of CLN3 RNA in a cell or animal, and in certain instances modulating the expression of CLN3 protein in a cell or animal. Such compounds, methods, and pharmaceutical compositions are useful to ameliorate at least one symptom or hallmark of a neurodegenerative disease. Such symptoms and hallmarks include poor motor function, seizures, vision loss, poor cognitive function, psychiatric problems, accumulation of autofluorescent ceroid lipopigment, brain tissue dysfunction or cell death, accumulation of mitochondrial ATP synthase subunit C, accumulation of lipofuscin, or astrocyte activation in brain tissue.
Abstract:
The present invention provides a method of diagnosing a neurodegenerative disease in a mammalian subject, preferably a human subject. The method comprises obtaining RNA from the mammalian subject, and assaying the RNA for an increase in the amount of ?fosB mRNA or in ratio of the amount of ?FosB mRNA to the amount of FosB mRNA as compared to that of a control. An increase in the amount of ?FosB mRNA or the ratio of the amount of ?FosB mRNA to the amount of FosB mRNA as compared to that of the control is indicative of the presence of the neurodegenerative disease.
Abstract:
The present disclosure relates generally to compounds comprising oligonucleotides complementary to a Leucine-Rich-Repeat-Kinase (LRRK2) RNA transcript. Certain such compounds are useful for hybridizing to a LRRK2 RNA transcript, including but not limited to a LRRK2 RNA transcript in a cell. In certain embodiments, such hybridization results in modulation of splicing of the LRRK2 transcript. In certain embodiments, such compounds are used to treat one or more symptoms associated with Parkinson's disease.