Engineered Fanzor2 (Fz2) nucleases with enhanced genome editing activity in mammalian cells and demonstrated genome editing in mice.
A high throughput, model-guided tool that prioritizes candidate amino acid substitutions for protein optimization under sparse data conditions.
Small cyclic peptides promote translation readthrough of premature termination codons, enabling fuller-length protein production from genes disrupted by nonsense mutations.
Intranasal delivery of dantrolene nanoparticles can improve motor neuron function, muscle strength, and movement coordination and can offer therapeutic benefits for treatment of Amyotrophic Lateral Sclerosis (ASL).
A flexible toolkit for human gene editing, activation, and repression that can be applied in orthogonally and in various disease models.
Technology allowing efficient formation, delivery, isolation, and enrichment of single-copy Human Artificial Chromosome (HAC) for use in research, plant production, gene and cell therapy and more.
Discovery and use of retina-specific promoters to rescue photoreceptor function specifically at clinically relevant, advanced stages of disease.
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Viral vector-mediated gene silencing of a novel short retinal isoform of Prolactin promotes photoreceptor survival.
Synthetic oligonucleotides blocking natural degradation of utrophin to treat muscular dystrophy.