ALS: From Genetic Complexity to Treatment Challenges and Advances
Amyotrophic Lateral Sclerosis (ALS), commonly called Lou Gehrig's disease, is a neurodegenerative disorder of the motor neurons that causes progressive muscle weakness and respiratory failure (1). ALS is the most common motor disease among adults (2), affecting around 60,000 people in the U.S. and ...
Nucleic Acid Therapeutics: Successes, Milestones, and Upcoming Innovation
Date: July 25, 2024 Time: 8am PDT / 11am EDT / 5pm CEST Title: Nucleic Acid Therapeutics: Successes, Milestones, and Upcoming Innovation Description: Nucleic acid-based therapies have become the third major drug class after small molecules and antibodies. The role of ...
A Nucleic Acid Approach to Antibody Therapeutics
Date: July 18, 2024 Time: 8am PDT / 11am EDT / 5pm CEST Title: A Nucleic Acid Approach to Antibody Therapeutics Description: Monoclonal antibodies represent a highly successful class of therapeutics that are used to treat complex diseases across a wide ...
Inherited Retinal Disorder: In Vivo CRISPR Therapy Provides Vision Improvements
Inherited retinal disorders (IRDs) are the leading cause of visual impairment, affecting approximately 1 in 2,000 people of all ages worldwide (1). There are many types of IRDs, each caused by a gene variant that affects how the retina functions, with one of the ...
Application of hydrophobically conjugated siRNAs for the treatment of dermatological and muscular diseases
Date: July 11, 2024 Time: 8am PDT / 11am EDT / 5pm CEST Title: Application of hydrophobically conjugated siRNAs for the treatment of dermatological and muscular diseases Description: This webinar will focus on siRNA delivery to skin and muscle using hydrophobic ...
President’s Pick Article – July 2024
Encapsulating decades of research, this article by Dr. Sudhir Agrawal, the 2022 OTS Lifetime Achievement Award recipient, explores the transformative potential of RNA-based therapies through the lens of over 30 years of learnings. The article highlights the remarkable progress made in the field, emphasizing ...
2023 OTS Paper of the Year Spotlight: A Framework for Individualized Splice-Switching Oligonucleotide Therapy
Date: August 22, 2024 Time: 8am PDT / 11am EDT / 5pm CEST Title: 2023 OTS Paper of the Year Spotlight: A Framework for Individualized Splice-Switching Oligonucleotide Therapy Description: Splice-switching antisense oligonucleotides (ASOs) could be used to treat a subset of ...
Annual Meeting Poster Awardee Spotlight
Date: June 27, 2024 Time: 8am PDT / 11am EDT / 5pm CEST Title: Functional Aptamer Selection for the Low-Complexity Domain of TDP-43 as a Potential Treatment Avenue for ALS Description: Targeting of TAR-DNA Binding Protein-43 (TDP-43), a protein implicated in ...
Angelman Syndrome Treatment Efforts and a Novel Prenatal Strategy
Imagine finding out your child may never walk, talk, feed, or dress themselves. Never have a job, get married, or have children. Never live independently. This long list of milestones never to be met is the experience for many parents with children diagnosed with ...
Branching into RNAi
Date: June 13, 2024 Time: 8am PDT / 11am EDT / 5pm CEST Title: Branching into RNAi Description: Our research focuses on the synthetic short-interfering RNAs (siRNAs) that silences the oncogenic mRNA expression of the Glucose Regulated Proteins (GRPs) leading to ...
