A Visual Guide to Gene Delivery - by Eryney Marrogi

A gene is the essential hereditary unit that passes traits from parents to offspring; a segment of DNA containing instructions for making a specific protein or molecule that performs a particular function in your body. And yet, despite being fundamental to our flourishing, genes can be broken.

Researchers have identified nearly 10,000 monogenic diseases, or conditions caused by errors in a single gene. Examples include hemophilia, which once plagued the royal houses of Europe, and sickle cell anemia, which affects around 8 million people worldwide.

Fortunately, as readily as these diseases are discovered and characterized, so too are many of their cures. As methods for resolving errors in DNA continue to advance, it has never been easier to fix a broken gene. Just this year, a baby with CPS1, a disease that causes ammonia to accumulate in the blood, became the first recipient of a CRISPR-tailored gene cocktail. The gene therapy is designed to protect this infant from the neurological disorders that otherwise would have resulted from this rare, and often fatal, condition. And while matching a gene therapy to its targeted mutation is an intricate and nuanced process, much boils down to two core elements: a vector and its cargo.

The first of these components is the therapeutic genetic payload, or cargo, which is designed to correct or regulate the disease-causing mutation, whether that means offering a healthy copy of a mutated gene or delivering precision-editing tools like CRISPR. Equally vital is the delivery vector, which carries this payload to specific cells.

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