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Patients Waiting

from The Rap Guide to Medicine by Baba Brinkman

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There are thousands of inherited diseases caused by mutations in key metabolic genes, of which just one is Niemann-Pick C. Patients are waiting for scientific progress in genetics and drug discovery to save them from their lethal inheritance. Bring on the CRISPR revolution!

lyrics

Somewhere in your body you’ve got a gene
Actually, trillions of copies of a gene
Every cell has a copy, it’s known as NPC
One, and it makes a protein
To be specific a 13-transmembrane
Endosomal cholesterol-binding protein
And if you’re asking how I know this is happening
Know this protein is involved in lipid trafficking
In your body, as part of the bricks and mortar
If it isn’t, you have a lysosomal storage disorder
Called Niemann Pick C, and you need help then
It makes you one in a hundred and fifty thousand
In Niemann Pick C, that gene is disabled
The protein is deficient and can’t deliver its payload
Cholesterol builds up like styrofoam
In a landfill, cloggin’ up the lysosome
Too many sphingolipids in the endocytic zone
Cells die, but Purkinje neurons die the most
So it’s known as childhood alzheimer’s
Neurodegeneration isn’t just for old timers
Kids born with Niemann Pick C
Decline cognitively and never live through their teens
So the race is on to find a treatment
Step one: trace the gene sequence

Look into genome, it’s a window
Compare species to get the info
Some people tell me that it’s sinful
I just wanna help my friends though

Patients waiting for some care
Peer into the darkness, never run scared
Answers come but it’s some here some there
Always somewhere

Somewhere in your body, every cell’s got a gene
NPC one, it makes a protein
And you can’t be healthy unless it’s intact
And that same gene has been identified in cats
And in cats, it makes a fat transporter
And you can give a cat a lipid storage disorder
Some would say that isn’t nice, those are kitty lives!
But if your kid is affected then the kitty cat dies
It gets spliced for scientific insights
But wait, that same gene has been located in mice!
And for some reason, a hundred dead mice
Just doesn’t seem as bad as a hundred dead cats
And why’s that? Why are dead kitties nastier?
Ever seen a knock-out mouse with ataxia?
It’s sad, I can’t imagine something awfuller
But luckily the same gene is also in drosophila
What a relief! When a fruit fly dies
Nobody cries, the room is full of dry eyes
But if you still feel for flies, hella sensitive
That’s fine! The gene is also in C. elegans
Worms, baker’s yeast, and toxoplasmosis
And in every one of them it triggers sphingolipidoses
Amazing, that means the gene has been conserved
Evolutionarily over a billion years

Look into the genome, it’s a window
Compare species to get the info
Some people tell me that it’s sinful
I just wanna help my friends though

Patients waiting for some care
Peer into the darkness, never run scared
Answers come, but it’s some here, some there
Always somewhere

Somewhere in a vial, on a tray in a lab
There’s a sample of a gene they’re injecting in a cat
In a mouse, in a fungus, in a worm, in a zebra fish
Which creates an amazing array of squeamishness
It’s funny how we feel increasing degrees of empathy
The closer we get to our own branch on the family tree
And I agree, use cats sparingly
Try to prioritize experiments on yeast
Besides, mammal generation times
Are too slow, you get more data from flies
And time flies, for some people the clock is ticking
Inside their neurons, cholesterol is sticking
But some cells are dying, while some are unaffected
And the answers why are coming from comparative genetics
When cholesterol is stuck it doesn’t get to where it’s needed
This knowledge is extremely helpful when designing treatments
Kinda like the knowledge that ebola virus binds 
To the same protein that NPC provides
And Niemann-Pick cells are immune to the virus
This research could even help us beat Alzheimers
And Parkinsons, ‘cause they have similar causes
Lysosome storage and lipid transfer processes
So there’s a twist to Niemann Pick C disease
Research on it is a gift to the species

Look into the genome, it’s a window
Compare species to get the info
Some people tell me that it’s sinful
I just wanna help my friends though

Patients waiting for some care
Peer into the darkness, never run scared
Answers come but it’s some here some there
Always somewhere

credits

from The Rap Guide to Medicine, released March 18, 2015
Lyrics by Baba Brinkman
Music by Mr. Simmonds
Scientific Consultation by Ethan Perlstein

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Baba Brinkman New York, New York

Science rapper and inventor of several novel hip-hop variants. Canadian transplant to New York. Pathological optimist.

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