Near Sequence Homology Does Not Guarantee siRNA Cross-Species Efficacy
Iris Valeria Rivera Flores, Kathryn Monopoli, Samuel Jackson, Dimas Echeverria, Daniel O’Reilly, Robert H. Brown, and Anastasia Khvorova Pages:234–244Published Online:27 August 2024 https://doi.org/10.1089/nat.2024.0030
Personalized ASO Provides Improvements for a Girl with KAND, an Ultra-rare Disease
Within the world of rare diseases exist conditions so sparse and infrequent they’re called nano-rare — or N of 1 — diseases and typically affect only one to thirty people worldwide (1). While less than 10% of rare diseases have approved treatments, nano-rare or ...
Angelman Syndrome Therapies Show Positive Results in Early Phase Clinical Trials
A clinical trial for a drug designed to treat the rare neurodevelopmental disorder known as Angelman syndrome (AS) has achieved a favorable safety profile and lessening of overall symptoms in trial participants. In a recent press release, Ionis announced the detailed HALOS study results ...
Encouraging Progress in the Hunt for a Huntington’s Treatment
Described as a combination of Alzheimer's disease, Amyotrophic lateral sclerosis, and Parkinson's disease all at once, Huntington's disease is a rare and debilitating neurological disorder passed down within affected families. With a 50% chance of a child inheriting it from a parent with the ...
Sequence- and Structure-Dependent Cytotoxicity of Phosphorothioate and 2′-O-Methyl Modified Single-Stranded Oligonucleotides
Authors: Laura V. Croft Mark Fisher, Tabassum Khair Barbhuiya, Serene El-Kamand, Samuel Beard, Aleksandra Rajapakse, Roland Gamsjaeger, Liza Cubeddu, Emma Bolderson, Ken O’Byrne , Derek Richard derek.richard@qut.edu.au, and Neha S. Gandhi Published Online: 17 June 2024 Abstract Single-stranded oligonucleotides (SSOs) are a rapidly expanding class ...
Nucleic Acid Therapeutics: Successes, Milestones, and Upcoming Innovation
Authors: Jillian Belgrad, Hassan H. Fakih, and Anastasia Khvorova Published Online: 3 April 2024 Abstract Nucleic acid-based therapies have become the third major drug class after small molecules and antibodies. The role of nucleic acid-based therapies has been strengthened by recent regulatory approvals and ...
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 ...
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 ...
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 ...
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 ...
