🧬 Rare Disease & Gene Therapy

Lentiviral Vector Integration-Site Genotoxicity SimulatorNewFire lentiviral gene-therapy vector batches into a 3D chromatin loop and watch where they… LNP Gene-Editing Delivery to HepatocytesNewInteractive 3D simulator of lipid-nanoparticle (LNP) delivery of gene-editing cargo to liver… AAV Empty/Full Capsid Density-Gradient SeparationNewSpin a mixed batch of empty and genome-full AAV capsids through a CsCl/iodixanol density… Accelerated Blood Clearance of PEGylated CRISPR LNPsNewInteractive 3D pharmacokinetic simulator of anti-PEG antibody-driven Accelerated Blood… Systemic AAV9 Gene Therapy: SMA Motor Neuron RescueNewModel the IV-infused AAV9 gene therapy used to treat spinal muscular atrophy: watch vector… AAV Episomal Dilution: Vector Genome Loss in a Growing LiverNewInteractive 3D hepatocyte-lobule simulator: AAV gene-therapy vector genomes stay episomal… AAV Vector Capsid EngineeringNewAdeno-associated virus vectors are re-engineered at the capsid surface to redirect tissue… ASO Splice ModulationNewAntisense oligonucleotide (ASO) modulation of alternative splicing involves the use of ASOs… Base & Prime Editing PrecisionNewBase editing is a precise genome editing technique that allows for the conversion of one… N-of-1 Ultra-Rare Disease Drug PipelineNewDevelopment of personalized antisense drugs for patients with unique mutations is a… Lysosomal Enzyme Replacement TherapyNewEnzyme replacement therapy for lysosomal storage diseases involves the administration of…