Synthetic Biology (in gene therapy context)
**What it means:** Synthetic biology is the field of engineering biological components and systems that don't exist in nature. In gene therapy, this includes designing custom viral vectors, optimizing...
Explainers
Plain-English explanations of MEF2C research terms, concepts, and scientific mechanisms.
AAV (Adeno-Associated Virus)
**What it means:** AAV is a tiny, harmless virus used as a **delivery vehicle** (vector) to carry therapeutic genes into cells. Think of it like a Trojan horse — the virus looks normal from the outsid...
Retinal (eye) in the context of gene therapy
**What it means:** The retina (the light-sensitive layer at the back of the eye) was actually the **first tissue** where gene therapy succeeded (Luxturna, 2017). The eye is "immune-privileged" — meani...