The Future of Biomedicine

Why is it important to study Biochemistry?

A complete understanding of disease, health and therapy requires vision and intuition at the molecular level

Discovery of new medical breakthroughs can be catalyzed by molecular insights

It’s not sufficient to know what powerful therapies are available – you need to understand whether they are appropriate

Linus Pauling and the dawn of “Molecular Medicine”

Not only was Pauling's group able to demonstrate that patients with sickle cell anemia have a different type of hemoglobin than healthy individuals, but also that blood taken from patients affected with the sickle cell trait, an asymptomatic form of the disease, contained a mixture of normal and defective hemoglobin in approximately equal amounts. They concluded that the sickle cell trait reflected a heterozygous condition, while sickle cell anemia reflected a homozygous one

Sickle Cell Disease

E6V is the most common mutation - reduces the charge on the chain at an exposed site on the surface - valine acts as hydrophobic patch to polymerize T state chains into a long tubular fibers

gene editing through directed double stranded breaks

scale from meganucleases to CRISPR/Cas9

CRISPR

clustered regularly interspaced short palindromic repeats

composed of an endonuclease protein whose DNA-targeting specificity and cutting activity can be programmed by a short guide RNA; known as a peculiar prokaryotic DNA repeat element for several decades before it was recognized as the bacterial immune system and subsequently harnessed as a powerful reprogrammable gene-targeting tool

CRISPR/Cas9 process

CRISPR structure

Non-homologous End Joining

The War on Chlorine Dioxide

practice medicine as a science… or be a “healer”

topical gel, sanitiser, cleans wood pulp - free radical

CRISPR popularity

exploded since 2012!

therapies since 2015!

What can be accomplished with CRISPR/Cas?

Enables introduction of DNA sequence changes that correct genetic defects in whole animals, such as replacing a mutated gene underlying liver-based metabolic disease

Allows DNA sequence changes in pluripotent embryonic stem cells that can then be cultured to produce specific tissues, such as cardiomyocytes or neurons, which could also eventually treat human disease.

Can replicate the genetic basis for human diseases in model organisms

Can change the DNA in the nuclei of reproductive cells that transmit information from one generation to the next (an organism's “germ line”).

Can thus alter the genetic makeup of every differentiated cell in an organism, ensuring that the changes will be passed on to the organism's progeny.

Changes to the human germ line could be made using this simple technology

CRISPR gene-editing for treating Sickle Cell Disease

It’s not about the technology – it’s about the patients

Victoria Gray, Brandon Williams, Jimi Olaghere

beginning of a new era in medicine

Emerging ethical dilemmas in biomedicine

EUGENICS - “it’s less about having your best baby, and more about being the best parent for your baby.”

germ-line editing

“premature,” “ethically problematic,” and even “monstrous.”

GMHs (genetically modified humans)

synthetic lifeforms

“frankenfoods”