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 ...
President’s Pick Article – June 2024
Another stellar review article summarizing the field now published in Nucleic Acid Therapeutics. The review in Nucleic Acid Therapeutics by Jillian Belgrad and colleagues discusses the significant progress and future potential of nucleic acid-based therapies (NATs). It highlights the historical milestones, including the approval of ...
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 ...
