Bridge Editing: A Novel Technique for Large-Scale DNA Modifications
LinkSource: Hiraizumi, M., Perry, N.T., Durrant, M.G. et al. Structural mechanism of bridge RNA-guided recombination. Nature 630, 994–1002 (2024).Gene editing has swiftly advanced since CRISPR was discovered, with CRISPR being developed not only to provide therapeutic gene editing treatments for humans but also for uses such as basic research, as a diagnostic tool, and for large-scale genetic screens, as well as in agricultural ...
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 ultra-rare diseases are often entirely excluded from drug development programs (1). However, the sphere of ...
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 for the multiple ascending dose (MAD) portion of the Phase 1/2 open-label study of ION582. ...
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 HD mutation, the fatal disease often leaves a devastating family legacy. In the United States ...
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 of therapeutics that comprises antisense oligonucleotides, microRNAs, and aptamers, with ten clinically approved molecules. Chemical ...
Bridge Editing: A Novel Technique for Large-Scale DNA Modifications
LinkSource: Hiraizumi, M., Perry, N.T., Durrant, M.G. et al. Structural mechanism of bridge RNA-guided recombination. Nature 630, 994–1002 (2024).Gene ...
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 ...
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 ...
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 ...
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 ...
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 ...
