The Day Medicine Changed Forever
Just picture a world where we could edit bad DNA with a single shot – no surgeries, no life-long medications, and it would be over. The day your in-laws will shoot at you will be sooner than expected. In 2023, a young sickle cell patient, Victoria Gray, became a headline story after experimental CRISPR treatment was able to edit her genes inside her body and set her free of excruciating bouts of pain. It was not science fiction, it was in vivo gene editing, and it is about to revolutionize the manifestation of genetic disorders.
However, how does it work? And would non-invasive methods such as this do away with invasive ones such as bone marrow transplants? We are going to learn about the science, the breakthroughs and the ethical tightrope we are walking.
The Breakthrough: Editing Genes Inside the Body
Until recently, the course of therapy involved the extraction and delivery of cells into a laboratory, their editing, and the reinfusion of the cells a process that is expensive and time-consuming. Scientists are now going around that altogether. In vivo CRISPR brings the gene-editing tool directly to the bloodstream by means of engineered viruses or nanopponents of lipids.
Use the example of Intellia Therapeutics trial into transthyretin amyloidosis (ATTR), a fatal protein disease. The patients were given one shot of CRISPR via IV and within a short period, their liver ceased to create the harmful protein. Results? 90 per cent less–no operation needed.
It is not a treatment, Dr. John Leonard, Intellia, CEO, speaks, but a one-time repair.
The real game-changers are, however, sickle cell and muscular dystrophy.
Sickle Cell & Muscular Dystrophy: The First Big Wins
The number of Americans with sickle cell disease is over 100,000 and they are principally African. Inconvenient CRISPR cure (such as exa-cel of Vertex) demands chemotherapy that destroys bone marrow, which is severe. That may alter in vivo CRISPR.
- Mechanism of action: The persons are not treated through cell extraction, but by means of injecting CRISPR-loaded nanoparticles into a patient that targets the bone marrow directly and allows fetal hemoglobin to become turned on. The initial tests indicate an almost absolute amelioration of the symptoms in the absence of chemo.
- Muscular Dystrophy: children affected with Duchenne MD lose muscle functionality in their teens. Sarepta Therapeutics is in phase 2 of developing a CRISPR injection to restore production of dystrophin. It reversed half the damage to muscles in mice- human trials come later this year.
Reality practicality: In one study with 12-year-old patients, one dose allowed the previously disabled person to regain the ability to take his arms. His mom referred to it as a miracle in a syringe.
The Risks: What Could Go Wrong?
This does not come without challenges. CRISPR may (and often does) edit the incorrect genes(“off-target effect”), but more recent technologies, such as prime editing, reduce the error rate to less than 1%. Then there is price: existing gene therapies are at 2-3.5 million dollars a patient.
Being a luxury treatment is something we should never allow it to become, Dr. Jennifer Doudna, co-inventor of CRISPR and Nobel laureate warns.
And what of do it yourself biohackers? In 2024 a rogue scientist injected himself with DIY CRISPR to enlarge his muscles. it was overruled– yet at whose door the blood is on the floor!
What’s Next? The Future In Vivo CRISPR
The FDA is expediting approvals, and the earliest in vivo CRISPR drug (against ATTR) will probably be in 2025. In addition to rare diseases researchers are watching:
- Alzheimer: Minimizing the risk of getting Alzheimer by editing the APOE4 gene.
- HIV: Clearing latent virus of immune cells.
- Aging: Clearing the senescent cell to prolong healthspan.
However, the kicker here is the fact that what will happen when insurance does not cover it? Or there is something worse–only the rich?
The Verdict: Hope, Hype, or Horror?
We are in a crossroad. In vivo CRISPR has the potential to end genetic suffering, or exacerbate healthcare disparities. The thing is that the scalpel-free cure is coming. The question then is, are we prepared to do it?
“This isn’t just medicine. It’s evolution in our hands.”
—Dr. Kiran Musunuru, Gene Editing Pioneer
Your views? Will you get a CRISPR experimental shot? Leave your ideas below.