🧬 Rare Disease & Gene Therapy

PEGylated LNP Antibody Clearance — 2D Pharmacokinetic SimulatorNew📱Interactive 2D pharmacokinetic simulator of anti-PEG antibody-driven Accelerated Blood… AAV9 Gene Therapy Dose-Response: SMA Motor Neuron Rescue (2D)New📱Interactive 2D dashboard of intravenous AAV9 gene therapy for spinal muscular atrophy (the… LNP Gene-Editing Delivery to Hepatocytes (2D)New📱A 2D top-down companion to the 3D LNP delivery simulator: the same two-stage probability… AAV Episomal Dilution: Vector Genome Loss in a Growing Liver (2D)New📱A 2D panable, zoomable hepatocyte-lobule grid: AAV gene-therapy vector genomes stay episomal… Lentiviral Integration Risk Track (2D)New📱A 2D linear genome-track map of lentiviral vector integration: watch falling capsids land on… AAV Empty/Full Capsid Density-Gradient Separation (2D)New📱A 2D isopycnic ultracentrifugation simulator: spin a mixed batch of empty and genome-full… Lentiviral Vector Integration-Site Genotoxicity SimulatorNew📱Fire lentiviral gene-therapy vector batches into a 3D chromatin loop and watch where they… LNP Gene-Editing Delivery to HepatocytesNew📱Interactive 3D simulator of lipid-nanoparticle (LNP) delivery of gene-editing cargo to liver… AAV Empty/Full Capsid Density-Gradient SeparationNew📱Spin a mixed batch of empty and genome-full AAV capsids through a CsCl/iodixanol density… Accelerated Blood Clearance of PEGylated CRISPR LNPsNew📱Interactive 3D pharmacokinetic simulator of anti-PEG antibody-driven Accelerated Blood… Systemic AAV9 Gene Therapy: SMA Motor Neuron RescueNew📱Model the IV-infused AAV9 gene therapy used to treat spinal muscular atrophy: watch vector… AAV Episomal Dilution: Vector Genome Loss in a Growing LiverNew📱Interactive 3D hepatocyte-lobule simulator: AAV gene-therapy vector genomes stay episomal… AAV Vector Capsid EngineeringNew📱Adeno-associated virus vectors are re-engineered at the capsid surface to redirect tissue… ASO Splice ModulationNew📱Antisense oligonucleotide (ASO) modulation of alternative splicing involves the use of ASOs… Base & Prime Editing PrecisionNew📱Base editing is a precise genome editing technique that allows for the conversion of one… N-of-1 Ultra-Rare Disease Drug PipelineNew📱Development of personalized antisense drugs for patients with unique mutations is a… Lysosomal Enzyme Replacement TherapyNew📱Enzyme replacement therapy for lysosomal storage diseases involves the administration of…