Treating Genetic Causes of Dravet Syndrome
In September 2014, identical twins Hudson Blake and Grady Lee were born and appeared seemingly perfect. However, when their mom went to return to work when the boys were 12 weeks old, she started to notice they both had an odd shiver. When Hudson ...
Antisense approaches for the treatment of Duchenne muscular dystrophy and recent advances to address DMD brain comorbidities
Date: May 23, 2024 Time: 8am PDT / 11am EDT / 5pm CEST Title: Antisense approaches for the treatment of Duchenne muscular dystrophy and recent advances to address DMD brain comorbidities Description: This presentation will cover the development of ASO-based approaches ...
2023 Annual Meeting Poster Awardee Spotlight: Stan and Moravčík
Date: May 16, 2024 Time: 8am PDT / 11am EDT / 5pm CEST Title: Validation lab: allowing standardized in vitro and in vivo experiments for candidate treatments for Duchenne muscular dystrophy Description: The Validation Lab was established in 2015 with support ...
Hope of New Treatments for Chronic Liver Disease MASH (NASH)
In the early stages of Metabolic Dysfunction-Associated Steatohepatitis (MASH), many patients are asymptomatic, though some may feel weak or fatigued or have an ache in their upper right abdomen. While lifestyle changes can help patients diagnosed early enough, more advanced stages of the disease ...
In-silico siRNA Off-Target Predictions: What Should We Be Looking For?
Date: May 2, 2024 Time: 8am PDT / 11am EDT / 5pm CEST Title: In-silico siRNA Off-Target Predictions: What Should We Be Looking For? Description: Assessing sequence-dependent off-target risks is a critical step during siRNA development to ensure the safety and ...
In vivo Optical Control of Gene-Silencing in Japanese Medaka Fish Embryos
Time: 8am PDT / 11am EDT / 5pm CEST Date: April 25, 2024 Title: In vivo Optical Control of Gene-Silencing in Japanese Medaka Fish Embryos Description: The siRNA oligonucleotide duplex inherent to biology, functions by binding and cleaving mRNA, a process ...
President’s Pick Article – April 2024
True to the vision of OTS, we worked with our journal publisher, Mary Ann Liebert publishers, Inc., through our BOD Committees to compile a list of important open-access Nucleic Acid Therapeutics (NAT) publications relating to the development of safe oligonucleotides. Finding high-quality publications to guide ...
RNA Single-base Editing Therapy that Treats Genetic Lung and Liver Disease Entered Clinical Trials
When Peggy's mom was in her forties, she started to have trouble breathing. Although she had never smoked, she had been exposed second-hand earlier in her life. Eventually, her mother ended up at the Mayo Clinic, where she was the 36th patient to be ...
Jennifer Doudna – Seeking to Improve the World with CRISPR
At 10:53 p.m. on Oct. 7, Jennifer Doudna woke to a buzzing sound. Picking up her phone — which she noticed had multiple missed calls and messages — she was greeted by the voice of Nature journalist Heidi Ledford. The day before, Doudna had ...
FDA Approved the First CRISPR Treatment – Potential Cure for People with Sickle Cell Disease
Victoria Gray was diagnosed with sickle cell disease (SCD) when she was just three months old; since then, episodes of pain and frequent hospitalizations have been a part of her life. Many of her dreams seemed like far-off impossibilities when the smallest things, like ...
