The most challenging thing
with sickle cell is understanding
that some days I might have up days
and some days I might have down,
as so your body.
This is what you use every day.
Having sickle cell, you're not allowed to,
I feel like enjoy some of the little things
that other people enjoy.
I feel like obviously we've been dealt
a crappy hand of cards and the goal is
to play to the best capabilities that you can.
Sickle cell anemia is a terrible disorder.
It is a hereditary disorder.
That is, it's something that occurs
in one's gene inherited from parents.
Worldwide, it's the most common hereditary disease.
Many millions worldwide, in Africa alone,
250,000 newborns are affected annually.
The hallmark of sickle cell disease is pain.
It happens when,
after the hemoglobin molecule,
which is specifically designed to bring oxygen
to our vital organs,
delivers the oxygen, the sickle mutation,
which is the change in the DNA,
creates this long strand,
making the round, flexible red blood
cell into a very sticky, stiff, sickle-shaped,
crescent-shaped red blood cell,
and that blocks blood flow.
When you don't get enough oxygen
to a tissue it causes pain.
Patients who have sickle cell disease experience pain.
They experience damage to vital organs where
blood flow can be altered by the red blood cells.
Lung problems.
When you don't get enough blood to your brain,
it causes a stroke.
These are all common complications
that affect the quality of life,
not just the lifespan in a person
with sickle cell disease.
I feel like I've experienced
some really crazy amounts of pain.
I've had both my lower extremities,
they had infarctions in them.
Imagine both your legs being broken at the same time,
it was the worst pain you can think of.
Then with my chest,
my pericarditis, I could barely breathe.
I was in constant pain all the time.
There's some days I just felt like a zombie.
We know that if you correct that mutation,
you take away the disease.
Anyone could imagine,
that if it was possible to correct
the gene in one's own cells,
they should be able to cure the disease.
When we first heard about CRISPR-Cas9,
we got very excited because
that could target the sickle gene,
correct the problem, and then,
restore healthy red blood cells in the circulation.
There are three groups of
scientists and clinical investigators working together
to accelerate the development of this technology
in humans with sickle cell disease.
One group at UCLA,
will be involved with manufacturing,
that is producing the blood stem cells
after correcting the sickle gene.
They are expert in that activity.
The group here in Oakland are experts
in hematology and sickle cell disease
and ultimately will lead a clinical trial
when we begin to treat patients.
Then the anchor I would say is the IGI,
the Innovative Genomics Institute at UC Berkeley,
that really brought that technology to us.
They are leading the effort and documenting,
at the DNA level
how many of these blood stem cells we've corrected.
We don't have enough therapies for sickle cell disease.
If it's related to societal pressures
and under-privileged,
under-represented populations in United States,
how rewarding would be to deliver
this amazing cure to this underserved population?
It's justice.
It's the first hereditary disease
ever defined in human beings,
primarily affects African Americans
and underserved population has difficult access
to health care in some cases.
Finally, we have a cure that
we can offer the majority of patients,
at least in our country.
That would be terrific.