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Investigation of Individualised Antisense Oligonucleotides (ASOs) in People With Unique Genetic Variants Causing Severely Debilitating, Life Threatening (SDLT) Central Nervous System (CNS) Conditions (EOM-MP1)

• Source: Investigation of Individualised Antisense Oligonucleotides (ASOs) in People With Unique Genetic Variants Causing Severely Debilitating, Life Threatening (SDLT) Central

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Pharmacokinetic Study of Oligonucleotides in Maixuekang Capsules

• Source: Exploring the Pharmacokinetics of Oligonucleotides in Maixuekang Capsules in Healthy Subjects

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Safety and Efficacy Study of Antisense Oligonucleotides in Duchenne Muscular Dystrophy

• Authors : Department of Health, United Kingdom; Sarepta Therapeutics, Inc.

• Source: Restoring Dystrophin Expression in Duchenne Muscular Dystrophy: A Phase I/II Clinical Trial Using AVI-4658Lu QL, Morris GE, Wilton SD, Ly T, Artem'yeva OV, Strong P, Partridge TA. Massive

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24 Month Open Label Study of the Tolerability and Efficacy of Inotersen in TTR Amyloid Cardiomyopathy Patients

• Authors : Rodney H. Falk, MD, Director, Brigham and Women's Cardiac Amyloidosis Program

• Source: 24 Month Open Label Study of the Tolerability and Efficacy of an Antisense Oligonucleotide (Inotersen) in Patients With Transthyretin (TTR) Amyloid Cardiomyopathy

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Safety, Tolerability and Pharmacokinetics of Multiple Ascending Doses of NIO752 in Progressive Supranuclear Palsy

• Source: A Randomized, Participant, Investigator and Sponsor Blinded, Placebo-Controlled Study to Evaluate the Safety, Tolerability and Pharmacokinetics of Multiple Ascending Doses of Intrathecally

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Safety and Pharmacokinetics of IMT504, an Immunomodulator and Tissue Repair Inducer (ECDA000/02)

• Authors : Immunalgia Therapeutics S.A.

• Source: Phase 1 Open-label Dose-escalation Study to Evaluate the Safety and Pharmacokinetics of IMT504 Phosphorothioate Oligonucleotide, an Immunomodulator and Tissue Repair Inducer, in Healthy Volunteers

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The Effect of an Antisense Oligonucleotide to Lower Transthyretin (TTR) Levels on the Progression of -Wild-type TTR Involving the Heart

• Authors : GlaxoSmithKline; Ionis Pharmaceuticals, Inc.; Rodney H. Falk, MD, Director, Brigham and Women's Cardiac Amyloidosis Program

• Source: An 18 Month Open Label Study Of The Tolerability And Efficacy Of An Antisense Oligonucleotide In Patients With Wild-Type Transthyretin Amyloid Cardiomyopathy (Senile Systemic Amyloidosis)

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Target of Suv420h1/2 in Hepatocytes

• Authors : Luca Valenti, Principal Investigator

• Source: Targeting the Epigenetic Regulators Suv420h1/2 in Hepatocytes to Treat Nonalcoholic Fatty Liver Disease

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Effect of PAE Hydrogel Loaded With tRF-ASO-Exo on Patients With Diabetic Ocular Surface Diseases

• Source: Effect of Poly（β-amino Ester）Hydrogel Loaded With tRNA-derived Fragments-antisense Oligonucleotides-exosomes on Patients With Diabetic Ocular Surface Diseases

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Human Liver ORganoids as a Model to Study the Development of Non-Alcoholic SteatOhepatitis (NASH) (REASON)

• Authors : Luca Valenti, Principal Investigator

• Source: Human Liver ORganoids as a Model to Study the Role of the I148M Variant of the PNPLA3 Gene in the Development of Non-Alcoholic SteatOhepatitis (NASH)

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