Targeting cancer stem cells with nanoparticle RNAi delivery to prevent recurrence and metastasis of ovarian cancer

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Research Objective Our objective is to develop novel treatments for women with ovarian cancer, specifically treatments that target stem cells to reverse drug resistance. These will treat recurrent metastatic disease.…

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Development of AS-241, an UNC13A Targeting Antisense Oligonucleotide (ASO) Treatment for ALS, for IND-enabling Studies

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Translational Candidate AS-241, an antisense oligonucleotide Area of Impact Amyotrophic Lateral Sclerosis Mechanism of Action AS-241 targets the cryptic exon (CE) of UNC13A and suppresses CE inclusion during RNA splicing,…

Continue ReadingDevelopment of AS-241, an UNC13A Targeting Antisense Oligonucleotide (ASO) Treatment for ALS, for IND-enabling Studies

Development of a Gene Therapy for Treatment of Guanidinoacetate Methyltransferase Deficiency-Translating In Vivo Proof of Concept to Support a Pre-IND

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Translational Candidate Adeno-associated viral vector serotyped for tropism to express guanidinoacetate methyltransferase in hepatocytes and brain cells. Area of Impact Developing a new therapy for Guanidinoacetate Methyltransferase Deficiency, where present…

Continue ReadingDevelopment of a Gene Therapy for Treatment of Guanidinoacetate Methyltransferase Deficiency-Translating In Vivo Proof of Concept to Support a Pre-IND