Glossary
Shared language for a changing field
A searchable glossary can be added later; for now the card structure and individual term pages are ready.
AAV
AAV, or adeno-associated virus, is a commonly used delivery vehicle for some gene therapies.
AI Drug Discovery
AI drug discovery uses machine learning and computational models to help find, design, or prioritize potential medicines.
ASO Therapy
ASO therapy uses short pieces of synthetic genetic material to change how RNA is processed or used by the cell.
Base Editing
Base editing is a gene-editing approach that can change certain DNA letters without cutting both strands of DNA.
Biological Foundation Model
A biological foundation model is an AI model trained on large biological datasets such as proteins, genes, cells, or molecular measurements.
Clinical Trial
A clinical trial is a research study in people designed to test whether an intervention is safe, useful, or both.
Digital Twin
A digital twin is a data-driven model that estimates how a patient, disease, organ, or trial might behave under different conditions.
Exon Skipping
Exon skipping is a strategy that tells cells to skip over a specific exon when processing RNA.
Gene Therapy
Gene therapy is a treatment approach that aims to add, replace, silence, or modify genetic instructions to treat disease.
ICV
ICV means intracerebroventricular, a delivery route into the fluid-filled spaces of the brain.
Intrathecal Delivery
Intrathecal delivery places medicine into the cerebrospinal fluid around the brain and spinal cord.
iPSC
An iPSC is an induced pluripotent stem cell, a lab-made stem cell created by reprogramming an adult cell.
Mosaicism
Mosaicism means not all cells in the body have the same genetic change.
Newborn Genome Sequencing
Newborn genome sequencing reads large parts of a baby's genetic code to look for disease-related variants early in life.
Phase I/II
Phase I/II trials combine early safety testing with early signals of dose, activity, or possible benefit.
Prime Editing
Prime editing is a gene-editing method designed to make targeted DNA changes without cutting both DNA strands.
RNA Therapy
RNA therapy targets RNA messages to change how proteins are made, reduced, increased, or processed.
Variant
A variant is a difference in DNA sequence compared with a reference genome.
X-Chromosome Inactivation
X-chromosome inactivation is the process by which most cells in people with two X chromosomes turn off one X chromosome.