Explainers
Clear guides to the science families are hearing about
Short video and article templates for gene therapy, ASOs, newborn genome sequencing, AI drug discovery, biological foundation models, and more.
What Is Gene Therapy?
Gene therapy is a treatment approach that tries to address disease at the level of the gene, often by adding, replacing, silencing, or editing genetic instructions.
What Is ASO Therapy?
ASO therapy uses short pieces of synthetic genetic material to change how RNA is read, processed, or reduced inside cells.
Gene Therapy vs ASO Therapy
Gene therapy and ASO therapy both work near the genetic root of disease, but they use different tools, timelines, and risk tradeoffs.
What Is Exon Skipping?
Exon skipping is a strategy that asks the cell to leave out a specific piece of RNA so it can make a shorter but potentially useful protein.
What Is a Vector?
A vector is a delivery vehicle used to carry a therapeutic payload into cells, often in gene therapy.
What Is Haploinsufficiency?
Haploinsufficiency means one working copy of a gene is not enough for the body to make the amount of protein it needs.
What Is Phelan-McDermid Syndrome?
Phelan-McDermid Syndrome is a rare genetic condition often linked to changes involving SHANK3 on chromosome 22.
What Is Rett Syndrome?
Rett syndrome is a rare neurodevelopmental disorder, most often linked to MECP2, that usually appears after early development seems relatively typical.
What Is Duchenne Muscular Dystrophy?
Duchenne muscular dystrophy is a progressive muscle disease caused by changes in the DMD gene that reduce or eliminate dystrophin.
Why Are Rare Disease Therapies So Hard?
Rare disease therapies are hard because the science, trial design, manufacturing, funding, and access challenges all arrive at once.
What Is Newborn Genome Sequencing?
Newborn genome sequencing looks for genetic conditions near birth, with the goal of finding actionable diagnoses earlier.
What Is AI Drug Discovery?
AI drug discovery uses machine learning and automation to help researchers find targets, design molecules, interpret biology, and prioritize experiments.
What Is a Biological Foundation Model?
A biological foundation model is an AI model trained on large biological datasets so it can learn patterns across genes, proteins, cells, or disease systems.
What Is a Digital Twin in Medicine?
A digital twin in medicine is a data-driven model that estimates how a patient, disease, organ, or trial might behave under different conditions.