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Printable Handouts
Navigable Slide Index
- Introduction
- Autoimmunity – the body at war with itself
- Autoimmune disorders and their incidence
- Type 1 diabetes (T1D)
- Diabetes type I
- T cell-mediated immune tolerance
- Regulatory T cells (Tregs)
- Mutations in the FOXP3 gene cause widespread autoimmune disease
- Polymorphisms in genes relevant for Treg function are associated with T1D
- Treg development
- Central and peripheral immune tolerance
- Treg-induced tolerance mechanisms
- Can Tregs be used as living therapeutics?
- Treg therapy for T1D in mice
- Antigen specificity is required for efficacy of Treg therapy for T1D in mice
- Polyclonal Tregs do not improve T1D (Phase 2, 110 patients)
- Financial disclosures
Topics Covered
- Autoimmunity
- Type 1 diabetes (T1D)
- T cell-mediated immune tolerance
- Regulatory T cells (Tregs)
- FOXP3 gene
- Central and peripheral immune tolerance
Links
Series:
- Gene Manipulation - How and Why?
- Periodic Reports: Advances in Clinical Interventions and Research Platforms
Categories:
Therapeutic Areas:
Talk Citation
Ferreira, L.M.R. (2026, April 30). Gene manipulation applications in autoimmune diseases 1 [Video file]. In The Biomedical & Life Sciences Collection, Henry Stewart Talks. Retrieved May 3, 2026, from https://doi.org/10.69645/FMNP8899.Export Citation (RIS)
Publication History
- Published on April 30, 2026
Financial Disclosures
- Dr. Leonardo M. R. Ferreira is an inventor in provisional and licensed cell and gene therapy patents, a consultant with Guidepoint Global and McKesson, and the founder and CEO of Torpedo Bio.
Gene manipulation applications in autoimmune diseases 1
Published on April 30, 2026
39 min
A selection of talks on Biochemistry
Transcript
Please wait while the transcript is being prepared...
0:00
Hi, everyone. My name
is Leo Ferreira.
I'm an Assistant Professor of
Pharmacology and Immunology at
the Medical University
of South Carolina and
the Hollings Cancer Center in
Charleston, South Carolina.
It's really a pleasure
to tell you today about
gene manipulation applications
in autoimmune disease.
0:21
We tend to think of
the immune system
as an army that protects us
from pathogens such as
viruses, bacteria, and cancer.
However, sometimes
the immune system
goes rogue and starts
attacking our own body.
It's like the body is
at war with itself.
This is manifested in
a group of diseases
called autoimmune diseases,
where the immune system
attacks our own tissues.
There are almost 100 different
autoimmune diseases.
You have psoriasis, rheumatoid
arthritis, vitiligo,
thyroiditis, multiple sclerosis,
Type 1 diabetes, and so on.
They are all characterized by
immune attack of
specific tissues.
Something about these
diseases is caused by
the immune system somehow losing
control and attacking itself.
1:13
While these autoimmune disorders
are relatively
rare in isolation,
if you combine almost
100 of them together,
they actually affect almost
one in 10 Americans,
which is a very large number
if you think about it.
People have studied the
etiology of these diseases,
how they're caused,
how long they go for,
how to make them better.
It's been extremely challenging
for most autoimmune disorders,
we do not know what causes them.
There are some theories.
It could be a viral
infection, could elicit them,
could be just some damage,
could be just pure bad luck.
There's, of course,
a genetic component,
but it's not perfect.
If you do twin
studies, for example,
and if one twin has
Type 1 diabetes,
only a third of the cases does
the other twin also
have Type 1 diabetes.
These are studies
where same genes,
same upbringing, same
environmental factors.
There's a lot of mystery
as to what causes
autoimmune disease and
what keeps it going.
But even then, what
I will argue during
this lecture is that we can
still treat and even cure
autoimmune disorders,
even if we don't always
know what caused them.
Type 1 diabetes is