Stuff to Read, Week 5
This week we talk CRISPR delivery, precision medicine anno 2030, gene editing equality and three-person babies arriving in the UK.
It’s 2030, and Precision Medicine has Changed Health Care – This is What it Looks Like
Imagine it is 2030. Ten-year-old Amy is wheeled into a children’s hospital clinic by her mother and, across town, 45-year-old Anh is visiting his oncologist one week after leaving hospital for his lung cancer operation.
Amy is slowly losing her ability to walk due to a muscle disorder that has only recently been given a name. Many diseases of muscle in children such as muscular dystrophy are caused by gene mutations (muscle disorders in adults are less likely to have a genetic mutation basis). In 2018, only around 40% of these mutations were known.
In 2030, we know 99% of mutations causing inherited muscle diseases worldwide. Because the costs of genetic sequencing are so much lower, more people have had their genomes scanned. Researchers have spent the last 20 years gathering information about various genetic markers and mutations into massive biobanks. This data is now globally accessible, which has led to international collaboration and better understanding of diseases.
So, 2030 is seeing a boom in precision medicine – where some diseases are defined more specifically than in the past with a focus on their molecular makeup. That is, the genes related to the DNA of the disease and other molecular elements. Precision medicine has vastly improved the ability to diagnose rare inherited diseases; to diagnose and treat cancers; and to aid in diagnosis and management of infectious diseases, dementia, heart disease and diabetes, among many others.
Doctors Select First Women to Have ‘Three-Person Babies’ in the UK
Doctors in Newcastle have been granted permission to create Britain’s first “three-person babies” for two women who are at risk of passing on devastating and incurable genetic diseases to their children.
The green light from the fertility regulator means that doctors at the Newcastle Fertility Centre will now attempt to make healthy embryos for the women by merging fertilised eggs created through standard IVF with DNA from female donors.
Gene Editing Needs to Be Available to Everyone
As technology changes, governments have to race to keep up with the changes — to manage them, to accommodate them, and to make sure their benefits are fairly distributed. Gene therapy is no different.
What Good is CRISPR if it Can’t Get Where it Needs to Go?
Your DNA is your body’s most closely guarded asset. To reach it, any would-be-invaders have to get under your skin, travel through your bloodstream undetected by immune system sentries, somehow cross a cell membrane, and finally find their way into the nucleus. Most of the time, that’s a really good thing. These biological barriers prevent nasty viruses from turning your cells into disease-making factories.
But they’re also standing between patients with debilitating genetic diseases and their cures. CRISPR, the promising new gene editing technology, promises to eradicate the world of human suffering—but for all the hype and hope, it hasn’t actually cured humans of anything, yet. Medical researchers have the cargo, now they just have to figure out the delivery route.
A Family’s Race to Cure a Daughter’s Genetic Disease
Personalised medicine promised a cure for rare genetic disorders. Now, patients and families themselves are trying to make up for its failures.
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